On March 14-15, the Massachusetts Biotechnology Council (“MassBio”) held its Annual Meeting in Cambridge, Massachusetts. The Meeting featured key topics such as biosimilars and a Keynote from John Crowley, Chairman and CEO of Amicus Therapeutics.
On the first day of the conference, Crowley exemplified many of the speakers’ entry in biotechnology, which originated with a family member or friend with a disease requiring development of a biotechnology product. For Crowley, it was his two children Megan and Patrick, were diagnosed with a severe neuromuscular disorder, Glycogen storage disease type II, known as Pompe’s disease. Rather that sitting still to wait for a cure, Crowley became involved in the process, first moving to Princeton, New Jersey, to be close to doctors specializing in the disease and leaving his job with Bristol-Myers Squibb. He later took a position as CEO of Novazyme Pharmaceuticals, a biotechnology research company located in Oklahoma City founded by Dr. William Canfield, which was conducting research on a new experimental treatment for the disease. Novazyme was acquired by Genzyme Corporation, which was then the world’s third largest biotechnology company. Crowley was put in charge of Genzyme’s global Pompe program, becoming the largest research and development effort in the company’s history.
Through these efforts, an experimental enzyme replacement therapy was developed, and Megan and Patrick Crowley received the therapy, which Crowley credits with saving his children’s lives. Crowley went on to become President and CEO of Orexigen Therapeutics and was named the President and CEO of Amicus Therapeutics, based in Cranbury, New Jersey, which he helped take public in 2007. Crowley’s efforts were documented in a Wall Street Journal article and other publications, which ultimately resulted in Harrison Ford working to bring the story to life in a major motion picture, Extraordinary Measures.
Crowley’s children are still alive and doing much better today at 15 and 16 years old. Crowley said the new therapy bought more time for him with his children, describing the biotechnology industry as “selling hope.” Crowley noted that today’s venture capital, however, is more risk averse today, because of the economic climate change. He said that risk taking requires persistence, and there is a constant need to innovate. From his own personal journey, Crowley said that he has learned the importance of humility.
On the second day, the Meeting featured a program on biosimilars. The panel was moderated by Kenneth L. Kaitin, Ph.D., Professor and Director, Tufts Center for the Study of Drug Development, Tufts University, and included Gino Grampp, Ph.D., Regulatory Policy Director, Amgen, and Jim Roach, M.D., Senior Vice President, Development and Chief Medical Officer, Momenta Pharmaceuticals, Inc. Roach said that Momenta hopes to build out more analytics following its successful model with enoxaparin and has been partnering with Sandoz and Baxter for new biosimilar products, some potentially interchangeable. Momenta hopes that FDA stays flexible in its approach with biosimilars and recognizes that the burden will be on the sponsor to overcome regulatory hurdles. And biosimilars are likely to be highly scrutinized, Roach added, potentially even by states, because no one wants to take any chances. Momenta, however, thinks this is good, because once biosimilars are approved, the public should expect that the approved products meet a high standard for approval and will be good.
Grampp gave “kudos” to Momenta for developing its analytics for enoxaparin, which was not so straight forward. Grampp predicted few companies would be able to file biosimilars and even fewer to obtain interchangeability. Grampp said Amgen has been meeting with FDA about biosimilars and has been pleased that FDA was seriously looking at their ideas for developing scientific standards. Grampp believed it that is important to keep the resources to implement this pathway and found the potential sequestration issues with user fees troubling (see Hamburg’s Keynote at MassBio here). Grampp said that Amgen feels “passionately” that post approval safety monitoring of biologics and biosimilars needs to be to a higher standard than for small molecules. When there are multiple drugs for the same condition, Grampp said, FDA should implement a mechanism to trace a particular product to a responsible manufacturer. Grampp said that Amgen supports a responsible, science-based pathway for biosimilars with patient safety, sound scientific analysis, and continued focus on the development of new therapies.
In response to questions about whether uncertainty in the biosimilars pathway from FDA is holding back potential filers, Roach did not think so. Roach thought that FDA’s broad guidances were better, because if industry asks for more guidance on interchangeability, for example, FDA could issue a very proscriptive guidance that is impossible to meet. Grampp believed that biosimilar development is limited more by the ecosystem of companies ready to develop biological products, which he believes will grow over time.
Some other interesting questions brought up the issue why Amgen has been supporting physician notification for biosimilar substitution at the state level. Grampp explained that Amgen’s goal was to create documentation for substitution for purposes of track and trace, especially when it comes to adverse events for latent effect such as immunogenicity. Amgen’s goal was not to scare away biosimilars–Amgen intends to develop biosimilars–but Amgen wants to have these controls in place for patient safety and confidence in biosimilars. To this, Roach commented that Momenta believes only interchangeable biosimilars should be substituted, but it would be going too far if patients had to sign informed consent for biosimilars, which has been proposed in some state bills.