Recently in Human Drugs Category

April 14, 2014

Zogenix Sues Massachusetts to Overturn Ban of Opioid Pain Drug

Thumbnail image for Thumbnail image for Thumbnail image for Thumbnail image for gavelgold.jpgZogenix, Inc., the San Diego-based manufacturer of the extended-release hydrocodone drug Zohydro ER, has sued the Governor of Massachusetts in U.S. District Court in Boston to overturn as unconstitutional the state's recent prohibition against prescribing and dispensing the medication.

Zohydro ER is the only FDA-approved hydrocodone drug indicated for daily, round-the-clock, long-term treatment of chronic pain for which other pain treatments are inadequate. The product, which was approved last year, is also the only available extended-release opioid drug containing hydrocodone alone, not combined with acetaminophen, which has been associated with liver damage.

The Governor of Massachusetts recently issued an "emergency declaration" establishing the ban, without consulting Zogenix, on the ground that a hydrocodone-only drug presents a greater risk of overdose and abuse than a hydrocodone combination drug. The ban would remain in place until "adequate measures are in place to safeguard against the potential for diversion, overdose and misuse."

Continue reading "Zogenix Sues Massachusetts to Overturn Ban of Opioid Pain Drug" »

February 27, 2014

FDA Adopts New Interpretation Awarding 5-Year NCE Exclusivity for Fixed Combination Drugs

multipills.jpgOn February 21, 2014, FDA issued a Draft Guidance that will now permit a fixed combination drug product containing a new active ingredient plus a previously approved active ingredient to qualify for New Chemical Entity (NCE) exclusivity, thereby preventing the filing of generic drug applications referencing the combination product for a period of 5 years. (A fixed combination drug is one containing more than one active ingredient, each in a fixed amount).

FDA's prior approach, in effect since 1994, had denied NCE exclusivity status to a fixed combination drug product that included an already-approved active ingredient. By virtue of the new Draft Guidance, the Agency is changing its interpretation of pertinent sections of the Federal Food, Drug, and Cosmetic Act and its own regulations. Going forward, FDA will determine NCE exclusivity by considering the newness of each drug substance (active ingredient) in a fixed combination drug product. If one active ingredient is new, NCE exclusivity can be awarded to the entire product.

As reasons for the change, FDA cites: (i) the emergence of combination drug treatment as a standard of care for serious diseases such as cancer, cardiovascular disease and infectious diseases (e.g., HIV), and (ii) the need to encourage the development of fixed combinations to treat these and other diseases, because particular combinations have been shown to improve treatment response, lower risk of resistance and lower rates of adverse events.

Continue reading "FDA Adopts New Interpretation Awarding 5-Year NCE Exclusivity for Fixed Combination Drugs" »

February 21, 2014

Prescription Drug TV Advertisements--How Do You Know Which Are Actually the Serious Drug Risks?

TVad.pngYou can hardly watch television without seeing a prescription drug advertisement. Often the most memorable part of the advertisement is the required voiceover disclosing a long list of all the risks associated with taking the drug. The problem becomes deciphering which risks are actually the serious ones. FDA seeks to find out if that long disclosure of risks results in "reduced consumer comprehension, minimization of important risk information, and potentially, therapeutic noncompliance due to fear of side effects."

On February 18, 2014, FDA issued a notice seeking comments about its proposed collection of information - "Disclosure Regarding Additional Risks in Direct-to-Consumer (DTC) Prescription Drug Television (TV) Advertisements (Ads)." FDA proposes to investigate the impact of limiting the risk disclosure in prescription drug television advertisements to only those that are "serious and actionable" plus an alert that there are other risks associated with the drug but which are not disclosed in the advertisement.

FDA would like to hear from you by April 21, 2014 about: whether you think its investigation is necessary "for the proper performance of FDA's functions;" whether the information will have practical utility; the validity of the methodology and assumptions its investigation will use; how the quality, utility and clarity of the information collected can be enhanced; and how the collection of information can be less burdensome on respondents.

Continue reading "Prescription Drug TV Advertisements--How Do You Know Which Are Actually the Serious Drug Risks?" »

February 19, 2014

Leerink's Global Healthcare Conference 2014 Focuses on a Variety of Emerging Health Products

leerink.pngOn February 12 and 13, 2014, Leerink Partners LLC ("Leerink") held its annual Global Healthcare Conference at the Waldorf-Astoria Hotel in New York, New York. Each year the Conference includes emerging themes in healthcare, where Leerink's equity analysts moderate discussions with MEDACorp specialists to provide unique and timely insights.

In addition to the company presentations, this year's line up featured the following panels or keynote speakers with some observed comments or trends:

• Panel: The Future of Medical Devices in an Evolving Landscape: A Shifting Emphasis to Patient Monitoring and Customizable Solutions

  • Patients view surgeons that incorporate robotics in their practice as the better doctors, driving more surgeons to utilize them in their practice. As surgeons become more familiar with these devices, patients may have more options for surgical procedures and implants.

  • Larger companies are looking at controlling infections caused by implanted medical devices with special coatings--either anti-infectives or antibiotics, particularly for use in higher-risk patients. There is an increasing need, however, for implants to have built-in tools for monitoring the devices. But as medical devices become more complex, such as hip, knee, or total joint replacements, these devices will require preapproval marketing applications ("PMAs") with clinical data rather than less costly and time-consuming 510(k)-type premarket clearance applications. Since PMAs cost companies more than 510(k) applications, these newer devices will cost third party payors and patients more.

  • Hospitals continue to be under a lot of pressure not to lose patients, so they may seek lower margins by having surgeons add anti-infective coatings or antibiotics rather than purchasing more costly versions with the coatings or by importing "generic" implants from other countries that may not be as rugged as the versions cleared for use in the U.S. FDA's new unique device identifiers and improved monitoring, however, may reduce use of such imported devices with unclear pedigrees.

Continue reading "Leerink's Global Healthcare Conference 2014 Focuses on a Variety of Emerging Health Products" »

January 3, 2014

Mylan Comments on Gilead's Stribild® Citizen Petition for Five-Year NCE Exclusivity

petition.pngIn a previous post, we covered Gilead's Citizen Petition to the FDA requesting FDA change its policy on how it allocates five years marketing exclusivity. Gilead argued that the current ruling whereby the five year exclusivity cannot be granted if even one active ingredient in the new drug application ("NDA") has been previously approved should be altered. Stribild®, which has two previously-approved active ingredients and two new active ingredients, is currently precluded from obtaining the five years new chemical entity ("NCE") exclusivity.

Mylan filed a Comment in Response, supporting FDA's current FDA interpretation, arguing against the various points raised in Gilead's Citizen Petition. First, Mylan points out that FDA's interpretation is not a matter of policy but governed by the plain language of the statute passed by Congress. The relevant statute is "The Drug Price Competition and Patent Term Restoration Act of 1984" ("Hatch-Waxman" or "the Act") which states in the section dealing with allocation of the five-year new chemical entity ("NCE") marketing exclusivity: "[I]f an application submitted under subsection (b) of this section for a drug, no active ingredient ... of which has been approved in any other application under subsection (b) of this section." Mylan argued that: (i) despite Gilead's attempts at re-interpreting the meaning of "drug" and "active ingredient", the statute still plainly says that there must be no active ingredient in the NDA that has been previously approved for the five year exclusivity to be granted and (ii) when Congress wrote "an application submitted under subsection (b) for a drug", it reasonably understood the word "drug" as used in this phrase to mean drug product and, not as Gilead would like to believe, a single component of the drug, such as the active ingredient.

As further support, Mylan pointed to the language of the three-year new clinical data marketing exclusivity provision:

Section 505(j)(5)(F)(iii) states: If an application submitted under subsection (b) for a drug, which includes an active ingredient (including any ester or salt of the active ingredient) that has been approved in another application under subsection (b), is approved after the date of the enactment of this subsection and if such application contains reports of new clinical investigations (other than bioavailability studies) essential to the approval of the application and conducted or sponsored by the applicant, the Secretary may not make the approval of an application submitted under this subsection for the conditions of approval of such drug in the subsection (b) application effective before the expiration of three years from the date of the approval of the application under subsection (b) for such drug. Thus, Mylan argued, taken together the plain language of the statute for both exclusivities leads to the conclusion that the current FDA interpretation is correct.

Continue reading "Mylan Comments on Gilead's Stribild® Citizen Petition for Five-Year NCE Exclusivity" »

November 18, 2013

Imbruvica® (ibrutinib) Approved To Treat Mantle Cell Lymphoma

Thelymphaticsystem_2011_large.jpgOn November 13, 2013, FDA approved Imbruvica® (ibrutinib) for the treatment of patients with mantle cell lymphoma ("MCL"), a rare and aggressive form of non-Hodgkin lymphoma or blood cancer. Imbruvica® will be co-marketed by Pharmacyclics, Inc. ("Pharmacyclics") based in Sunnyvale, Calif. and Janssen Biotech, Inc. ("Janssen") based in Raritan, N.J and will be sold as a single agent oral capsule.

Imbruvica® has been approved for patients with MCL who have received at least one prior therapy and works by blocking a specific protein called Bruton's tyrosine kinase ("BTK"). Non-clinical studies have shown that blocking BTK inhibits malignant B-cell survival. The recommended dose of Imbruvica® is 560 mg (four 140 mg capsules) once daily. About six percent of all non-Hodgkin lymphoma cases in the U.S. are classified as MCL. MCL is often diagnosed in an advanced stage after it has already spread to the lymph nodes, bone marrow and other organs.

Imbruvica® was approved as part of the Breakthrough Therapy Designation ("BTD") program that facilitated its development, review, and approval. Imbruvica® was granted three BTDs by FDA, including relapsed or refractory MCL, and is the second drug with a BTD to receive FDA approval.

Continue reading "Imbruvica® (ibrutinib) Approved To Treat Mantle Cell Lymphoma" »

November 13, 2013

Compounding Pharmacy Bill Advances in Senate (Updated Again - Signed Into Law)

mortar and pestle.jpg

[Update: After unanimously passing the Senate on November 18, 2013, H.R. 3204--the Drug Quality and Security Act--is set to become law after an expected signature from President Obama. President Obama signed into law on November 27, 2013.]


On November 12, 2013, the Senate voted to close debate on and advance a compounding pharmacy bill--H.R. 3204--aimed at tightening government oversight of pharmacy compounding and creating a national tracking system for prescription drugs. The 97-1 vote indicated overwhelming bipartisan support for the proposal, which passed the House in September. The lone dissenter, Sen. David Vitter, objected to a final Senate vote on the proposal because he wanted the Senate to first vote on a measure that he proposed to require lawmakers to disclose which of their aides are signing up for health insurance under the Affordable Care Act and which are remaining in the Federal Employee Benefit Program.

Originally introduced in the House by Rep. Fred Upton, H.R. 3204--the Drug Quality and Security Act--seeks to address two major concerns regarding the safety and quality of the U.S. drug supply. First, and in response to last year's deadly meningitis outbreak, the Bill seeks to strengthen government oversight of compounding pharmacies. More specifically, Title I--the Compounding Quality Act--would amend the Federal Food, Drug, and Cosmetic Act ("FD&C Act") as to the regulation of compounding pharmacies, which have historically been regulated by state pharmacy boards. Under the proposal, pharmacies that perform traditional, small-scale compounding will continue to be regulated by state pharmacy boards. Additionally, drugs compounded by or under the direct supervision of a licensed pharmacist in a facility can elect to register as an outsourcing facility. In doing so, these drugs would be exempt from the FD&C Act's labeling, new-drug, and proposed track-and-trace requirements if certain conditions were met, including: (1) proper registration of the facility; (2) no compounding using bulk drug substances; (3) compounding with ingredients that comply with applicable standards; (4) no compounding of drugs that have been withdrawn or removed due to lack of safety or efficacy; (5) no compounding of drugs that are essentially a copy of one or more approved drugs; (6) no compounding of drugs that have been identified as ones that present demonstrable difficulties for compounding; (7) no sale or transfer of the compounded drug by any entity other than the outsourcing facility; and (8) appropriate labeling.

The Bill also seeks to increase protection of the prescription-drug supply chain. More specifically, Title II--the Drug Supply Chain Security Act--will establish requirements to establish a track-and-trace system that will follow prescription drugs from manufactures to retail pharmacies. This national system should facilitate greater protection against counterfeiting, earlier detection of possible drug shortages, simpler recalls of defective drugs, and other drug supply-chain issues. While there will be certain waivers and exemptions, the proposal would require drugmakers to affix a product identifier on each package and case of product intended to be introduced in a transaction into commerce. Ten years after enactment, the Bill adds an electronic-tracing requirement. The Bill will require FDA to publish guidelines: (1) establishing standards for the interoperable exchange of transaction information, transaction history, and transaction statements; and (2) establishing the process by which companies may obtain waivers, exceptions, and/or exemptions to the product-identifier requirements. The national track-and-trace program will also preempt all state and local requirements regarding tracing drugs through the supply channels.

Continue reading "Compounding Pharmacy Bill Advances in Senate (Updated Again - Signed Into Law)" »

November 12, 2013

Aptiom® (Eslicarbazepine Acetate) Approved for Partial-Onset Seizures

epilepsy.jpgOn November 8, 2013, FDA approved Aptiom® (eslicarbazepine acetate), manufactured by Sunovion Pharmaceuticals ("Sunovion"), as a once-daily, immediate-release, adjunctive therapy for partial-onset seizures associated with epilepsy in adults 18 years or older. Partial or localized seizures affect only part of the brain at onset and are the most common type of seizure in epilepsy patients. Seizures can cause a wide range of symptoms, including repetitive limb movements, unusual behavior, and generalized convulsions with loss of consciousness and can have serious consequences, including injury and death. Epilepsy is one of the most common neurological disorders affecting nearly 2.2 million people in the U.S.

Aptiom® is an oral drug that stabilizes the inactive state of voltage-gated sodium channels and blocks T-type voltage-gated calcium channels. Sunovion recommends treatment using 400 mg once daily initially for one week followed by a dosage increase to a recommended maintenance dosage of 800 mg once daily. The maximum recommended maintenance dosage is 1,200 mg once daily. FDA noted that existing treatments do not achieve satisfactory seizure control from existing treatments. Sunovion, the U.S.-based unit of the Japanese drug maker Dainippon Sumitomo Pharma Co., expects Aptiom® to be available in U.S. pharmacies in the second quarter of 2014. FDA did not classify Aptiom® as a controlled substance.

Sunivion submitted Aptiom® for approval based on three, large phase 3 randomized, double-blind, placebo-controlled, safety and efficacy trials that included more than 1,400 patients with partial-onset seizures inadequately controlled by one to three concomitant, antiepileptic drugs, including carbamazepine, lamotrigine, valproic acid, and levetiracetam. These trials, jointly performed with BIAL-Portela & Ca, S.A. ("BIAL"), demonstrated a statistically-significant reduction in standardized seizure frequency compared to placebo. Significantly more treated patients had a seizure frequency reduction of 50% or more from baseline (41% compared to 22%).

Continue reading "Aptiom® (Eslicarbazepine Acetate) Approved for Partial-Onset Seizures " »

November 8, 2013

Korea-Maryland USA Bio Expo Held in Rockville, Maryland Last Week

Korea-MD, USA Bio Expo Opening Ceremonies.JPGOn November 7 and 8, 2013, FLH attorneys Brian J. Malkin, Charlie J. Raubicheck, and Scot B. Pittman attended and participated as sponsors of the 3rd Annual Korea-Maryland, USA Bio Expo. The Expo hosted leading Korean pharmaceutical, biotechnology, and medical device companies at the Universities at Shady Grove Conference Center in Rockville, Maryland. The event provided networking opportunities and seminars from industry professionals with the goal to encourage collaboration of Korean biotechnology businesses utilizing the resources in the Washington, D.C. area.

The opening ceremonies featured presentations by a variety of local and Korean representatives committed to helping Korean business set up operations in the United States, particularly Maryland, and specifically Montgomery County. The speakers included:


  1. Ambassador for Green Growth & Environment, Kyungryul An

  2. Embassy of the Republic of Korea, Consul-General Do-ho Kang

  3. Maryland Secretary of State, John P. McDonough

  4. BioMaryland Center Executive, Director Judy Britz

  5. Montgomery County Executive, Isiah Leggett

  6. Montgomery County Department of Economic Development Director, Steve Silverman

  7. Hanbat National University Graduate School, Dean Byung Wook Ahn

  8. George Washington University, Department Chair of East Asian Languages and Literatures Young-Key Kim-Renaud.

The Maryland representatives highlighted some of the advantages for doing business in Maryland. For example, Maryland offers income tax credits equal to 50% of an eligible investment for investors in Qualified Maryland Biotechnology Companies ("QMBCs"). This tax credit program offers incentives for investment in seed and early stage, biotech companies, up to $250,000. To qualify, companies are required to: be less than 15 years old; have their headquarters and base of operations in Maryland; employ fewer than 50 people, and have a valid certification from the Department of Business and Economic Development ("DBED"). Investors are required to submit applications prior to making an investment. DBED reviews the applications and issues initial credit certifications within 30 calendar days.(http://www.choosemaryland.org/businessresources/pages/biotechnologyinvestmenttaxcredit.aspx ). Chevy Chase, Maryland also is home to a branch of NEA, one of the country's largest venture capital funds, with the potential to help support Maryland businesses such as those in the biotechnology field.

Continue reading "Korea-Maryland USA Bio Expo Held in Rockville, Maryland Last Week" »

October 9, 2013

Bayer's Adempas® Pulmonary Hypertension Drug Receives FDA Approval

heart.jpgOn October 8, 2013, FDA published a press release announcing its approval of Bayer HealthCare Pharmaceuticals, Inc.'s ("Bayer's") Adempas® (riociguat) to treat two types of Pulmonary Hypertension ("PH"): (1) chronic thromboembolic pulmonary hypertension ("CTEPH") and (2) pulmonary arterial hypertension ("PAH"). PH is an increase in blood pressure of the pulmonary arteries. Normal pressure in the pulmonary arteries is 8-20 mm Hg and PH occurs when the pressure is 25 mm Hg or higher. The World Health Organization ("WHO") has divided PH into five groups. Group 1 (PAH), Group 2 (PH with left heart disease), Group 3 (PH associated with lung disease), Group 4 (PH caused by blood clots (includes CTEPH)), and Group 5 (PH caused by other diseases). Adempas® is the first drug approved in the U.S. to treat two types of PH, specifically Groups 1 and 4.

"Adempas is the first in its drug class approved to treat pulmonary hypertension and the first drug of any class to be shown to be effective for patients with CTEPH," said Norman Stockbridge, director of the Division of Cardiovascular and Renal Drug Products in the FDA's Center for Drug Evaluation and Research ("CDER"). In CTEPH, blood clots travel from the body and clog the pulmonary arteries, forming more clots and increasing resistance to blood flow. In PAH, the pulmonary arteries become narrowed or tightened restricting blood flow. CTEPH and PAH both cause the heart to work harder to pump blood through the lungs, and this increased strain often causes shortness of breath, fatigue, weakness, and sometimes heart failure.

Adempas® is a guanylate cyclase stimulator which catalyzes the synthesis of cGMP, the signal for relaxation of smooth muscle. Relaxation of smooth muscle causes vasodilation, increased blood flow, and reduced blood pressure. The standard treatment for CTEPH is a complex surgery to clear the clots from the arteries, but for patients with inoperable CTEPH or recurrent CTEPH after surgery, Adempas® is the first and only FDA-approved drug treatment. FDA approved Adempas® with the goal of helping affected patients increase their ability to exercise.

Continue reading "Bayer's Adempas® Pulmonary Hypertension Drug Receives FDA Approval" »

September 27, 2013

Strategic Scientific Business Tactics Explored at MassBio Forum in Cambridge

biotechnology.jpgOn September 26, the Massachusetts Biotechnology Council ("MassBio") hosted the fourth part of its "Adventures in Biotech" Forum series entitled, "Executing the Dream II: Eyes on the Price - Strategic Science Tactics & the Pursuit of Business Objectives" . The purpose of this Forum was to discuss ways that scientific strategy may be worked into the philosophy of a new company, how the scientific strategy can propel the growth of the company, and how the scientific strategy may change over time as a result of that growth. The speakers included Birgit Schoeberl, Vice President, Early Stage Discovery, Merrimack Pharmaceuticals ("Merrimack"); Muthiah Manoharan, Ph.D., Senior Vice President Drug Discovery, Alnylam Pharmaceuticals ("Alnylam"); Laurence Reid, Ph.D., Senior Vice President, Chief Business Officer, Alnylam; and Steven Tregay, Ph.D., Founder, President and Chief Executive Officer, Forma Therapeutics ("Forma").

Each of the speakers had a different approach as to how they incorporated their scientific strategy into their business. Tregay, for example, described Forma as a "sustainable research and development discovery engine" focused on oncology therapies that can screen 30-40 individual (not combinational) targets a year using an iterative process that includes: computational and medicinal chemistry, parallel synthesis, X-ray crystallography and relevant biology studies. According to Tregay, Forma's science permits it to mitigate risk by running in parallel a large number of discovery programs while continuously assessing the molecules, prioritizing targets for their pipeline at the appropriate scale, and maintaining communication between all scientists, and project teams. As part of its strategy, Forma first goes broad to investigate a cancer target technology and then more specific as aspects of that technology are validated and ready for candidate screening. Integral in Forma's strategy is forming partnerships with companies interested in screening many potential cancer candidates quickly rather than few candidates exhaustively, where it already has formed partnerships with Celgene, Boehringer Ingelheim, Genentech, and Johnson & Johnson, as well as some research universities.

Reid explained that Alnylam's focus has been on building its platform technology on ribonucleic acid interference ("RNAi"), a natural mechanism for silencing specific genes. RNAi is a biological process in which ribonucleic acid ("RNA") molecules inhibit gene expression, typically by causing the destruction of specific messenger RNA ("mRNA") molecules. RNAi has gone by other names in the past, including co-suppression, post transcriptional gene silencing ("PTGS"), and quelling. Genes provide cells with the instructions for making proteins, and abnormal proteins are frequently the cause of human disease. The theory behind RNAi therapy, therefore, is to use double-stranded RND ("dsRNA") to silence certain genes involved in the diseased state and reduce the occurrence of the associated disease. Scientists at Alnylam developed a new strategy to trigger RNAi in mammalian cells using relatively-small dsRNAs--long enough to induce RNAi, but small enough to avoid inducing an immune response--to permit RNAi to be considered as a new therapeutic strategy.

Continue reading "Strategic Scientific Business Tactics Explored at MassBio Forum in Cambridge" »

September 23, 2013

505(b)(2) NDA RLD and Impurities Explained in Petition Response

Thumbnail image for ANDA drugs.jpgOn September 18, 2013, FDA provided a response to two citizen petitions filed by David B. Clissold, Director, Hyman, Phelps & McNamara P.C. ("Clissold's Petitions"). Clissold's Petitions requested that FDA refuse to file any new drug application ("NDA") filed under section 505(b)(2) of the Federal Food, Drug, and Cosmetic Act ("FD&C Act") (hereafter, "505(b)(2) NDA") seeking approval of a buprenorphine/naloxone drug product in the form of a polymer film for oral mucosal membrane absorption unless the application referenced NDA No. 22-410 for Suboxone® sublingual film (buprenorphine hydrochloride / naloxone hydrochloride) ("Suboxone Film"). As a quick summary, a 505(b)(2) NDA is not identical (i.e., generic) to a previously-approved drug. Instead, a 505(b)(2) NDA contains full reports of safety and effectiveness where at least some of the information required for approval came from studies that the applicant did not conduct/sponsor or have a right to reference. Clissold's Petitions also asked that FDA refuse to approve such application unless any genotoxic or potentially genotoxic impurity associated with naloxone are appropriately limited. FDA granted and denied in part Clissold's Petitions following a review of the Petitions, a supplemental letter from Clissold, and additional filed comments from a 505(b)(2) NDA applicant for buprenorphine/naloxone buccal film.

Following a discussion of the 505(b)(2) NDA approval framework, FDA agreed that it could refuse to file certain 505(b)(2) NDAs that did not "on its face contain required information" yet declined to so hold for the buprenorphine/naloxone product in question, if it failed to reference Suboxone Film. Like Clissold, FDA referenced its 2004 citizen petition response to Abbott Laboratories and Laboratories Fournier regarding fenofibrate ("Fenofibrate Petition"). In FDA's response to the Fenofibrate Petition FDA explained that when there is no pharmaceutical equivalent to a listed drug, an applicant should submit its 505(b)(2) NDA referencing the most similar product to reduce the amount of additional data to submit for approval.

FDA further explained in its response to Clissold's Petitions, however, that while an applicant's failure to reference the most similar product would not be a basis for a refusal to file, it could become an approval issue, if the applicant cited to an inappropriate reference product. In essence, an applicant's "approach to its development program" should be the guide to the appropriate reference product. And if there were a pharmaceutically-equivalent product, then the 505(b)(2) NDA applicant should identify such product and file the appropriate patent certifications. This latter situation generally describes a situation where a 505(b)(2) NDA may be submitted instead of an abbreviated new drug application ("ANDA") following approval of a suitability petition for certain allowed changes to previously-approved NDA products. Some of the situations where a suitability petition may be submitted are for ANDAs that differ from the listed drug in route of administration, dosage form, strength, or if it contains a different active ingredient in a combination product.

Continue reading "505(b)(2) NDA RLD and Impurities Explained in Petition Response" »

September 20, 2013

HHS Adverse Event Plan Mentions FDA but Focuses on CMS as a Central Actor

HHSOn September 4, 2013, the Department of Health and Human Services ("HHS") released a report entitled "National Action Plan for Adverse Event Prevention" ("Plan"). The 176-page report purports to "engage all stakeholders in a coordinated, aligned, multi-sector, and health literate effort to reduce ADEs [adverse drug events] that are most common, clinically significant, preventable, and measurable." For this effort, the Plan focused on the intended end-users, "policymakers, health care professionals, public and private sector organizations, and communities who can organize and take action toward preventing high-priority ADEs." The high priority targets identified to prevent ADEs are the drug classes of anticoagulants, diabetes agents, and opioids. The four-pronged approach includes: surveillance, prevention, incentives and oversight, and research.

ADEs may occur in both inpatient (hospital) and outpatient and long-term settings. According to statistics cited in the Plan, ADEs account for about two million hospital stays annually and prolong hospital stays by about 1.7 to 4.6 days. ADEs in outpatient settings account for reportedly over 3.5 million emergency department visits, resulting in 125,000 hospital admissions each year.

The Plan acknowledges that a certain amount of passive (voluntary) reporting of ADEs comes via FDA's Adverse Event Reporting System ("FAERS") as well as FDA's Sentinel Initiative that monitors over 125 million lives but, the Plan says, "which do[es] not constitute a nationally representative sample, but for specific studies, FDA's Sentinel Initiative has the potential to access health records to confirm coded data or provide additional data." FDA's Sentinel Initiative was established in response to the FDA Amendments Act ("FDAAA") passed in 2007, which mandated that FDA establish an active surveillance system for monitoring drugs, using electronic data from healthcare information holders. The goal of the Sentinel Initiative is a new active surveillance system that could be used to monitor all FDA-regulated products. In particular, the Sentinel System draws on existing automated healthcare data from multiple sources to actively monitor the safety of medical products continuously and in real time.

Continue reading "HHS Adverse Event Plan Mentions FDA but Focuses on CMS as a Central Actor" »

September 19, 2013

House Compounding Bill Seeks to Provide Clarity and Balance Between FDA and State Boards of Pharmacy Oversight

pharmacymortarpestle.pngLast week, Representatives Morgan Griffith (R-Virginia), Diana DeGette (D-Colorado), and Gene Green (D-Texas) introduced in the House compounding legislation entitled the "Compounding Clarity Act of 2013" (H.R. 3089) that could give FDA additional official authority under the Federal Food, Drug, and Cosmetic Act ("FD&C Act") over compounding facilities. The Bill comes after almost a year after contaminated drugs from a Massachusetts compounding facility caused 64 people to die from fungal meningitis.

According to Green, "For the last several weeks, a bipartisan and bicameral group of lawmakers have been meeting to make sure we never relive the tragedy of the 2012 fungal meningitis outbreak that originated in a compounding pharmacy in Massachusetts." Griffith said that the bill would "prevent another [New England Compounding Center]-type outbreak from occurring and ensuring the quality and safety of all compounded drugs in the country."

The Bill would amend Section 503A of the FD&C Act with a new framework and standards for traditional compounding pharmacies and additional FDA oversight for large-scale compounding like the New England Compounding Center. The legislation would help remove the Constitutional concerns concerning advertising that had been raised in various court cases and had raised certain questions about FDA's regulatory authority over compounding.

Continue reading "House Compounding Bill Seeks to Provide Clarity and Balance Between FDA and State Boards of Pharmacy Oversight" »

July 18, 2013

Gilotrif® with Therascreen EGFR PCR Kit Approved for Lung Cancer Treatment

lung-cancer-500x360.jpgFriday, FDA approved Boehringer Ingelheim's drug, Gilotrif® (afatinib), to treat patients with late-stage metastatic Non-Small Cell Lung Cancer ("NSCLC"). Gilotrif® is a tyrosine kinase inhibitor that inhibits cancer-causing proteins. NSCLC is any type of epithelial lung cancer other than small cell lung cancer ("SCLC"). The most common types of NSCLC are squamous cell carcinoma, large cell carcinoma, and adenocarcinoma. Although NSCLCs are associated with cigarette smoke, adenocarcinomas may be found in patients who have never smoked. As a class, NSCLCs are relatively insensitive to chemotherapy and radiation therapy compared with SCLC.

Gilotrif® targets specific proteins caused by epidermal growth factor receptor ("EGFR") gene mutations, which are present in about 10 percent of NSCLC patients. Specifically, Gilotrif® targets proteins expressed by EGFR exon 19 deletions and exon 21 L858R substitutions. The drug is being approved concurrently with a companion diagnostic kit that helps determine if a patient's lung cancer cells express EGFR mutations. The kit is called therascreen EGFR RGQ PCR Kit, and is made by Qiagen N.V.

FDA approval of Gilotrif® for NSCLC is a big deal. Lung cancer is the number one cause of cancer-related deaths and NSCLC makes up about 85% of all lung cancers. An estimated 160,000 people will die in the U.S. from lung cancer this year, according to the National Cancer Institute, and 1.38 million deaths worldwide are attributable to lung cancer. Lung cancer is the cause of 18% of all cancer deaths.

Continue reading "Gilotrif® with Therascreen EGFR PCR Kit Approved for Lung Cancer Treatment" »