Two of the most noteworthy drugs to win FDA approval over the past year, eteplirsen (Exondys 51) and nusinersen (Spinraza), have something important in common: Patient groups have played a critical role in both of their stories.
Eteplirsen, Sarepta Therapeutics’ (NASDAQ: SRPT) Duchenne muscular dystrophy drug, was a test case for the increasing power of patient advocacy. An FDA advisory committee hearing on eteplirsen last year saw throngs of patients and advocates pleading with regulators to approve the drug despite flawed data. Though the panel of outside experts recommended against eteplirsen, the FDA showed flexibility and later approved it anyway, a nod to the greater power the patient community had, and the pressure it put on the agency.
As Xconomy reported last year, advocates for patients with spinal muscular atrophy were watching the Duchenne proceedings closely and learning from their counterparts as Biogen’s (NASDAQ: BIIB) nusinersen neared the market. They were involved in the education of regulators about SMA, and, since nusinersen’s approval, have relentlessly fought insurers for access to the expensive drug. Biogen reported higher than expected sales this past week, and CEO Michel Vounatsos specifically pointed to “motivated families, parents, [and] patient advocacy groups” as a big reason why.
Both of these stories—and the implications they, and more broadly speaking, mobilized patient communities have for drug development—will take center stage at Xconomy’s latest event, “What’s Hot in Boston Biotech,” at Biogen’s headquarters in Cambridge, MA, on May 11. We’ve brought together a diverse group of speakers to hold interactive chats on a variety of big topics affecting the life sciences in Boston and beyond, from the challenges of company building to the opioid crisis, immigration, and the future of cancer care. You can check out the lineup and the agenda here.
There are just two weeks remaining before the event, but you’ve got one last shot at a discount if you grab tickets here. Hope to see you all soon at Biogen.