It’s tempting to interpret the Food and Drug Administration’s surprise rejections of drugs from Gilead Sciences and Biomarin Pharmaceuticals this week as an agency-wide sentiment downshift, raising concerns that fewer new medicines will reach the market.
Don’t make that mistake.
The overall numbers refute the “FDA is turning conservative” thesis. Through eight months of this year, the FDA has approved 36 new drugs, both small molecules and biologics, according to a tally kept by Ira Loss of Washington Analysis, a regulatory research firm.
There are another 30 to 40 “new” drugs (depending on how you define the term) under review at the FDA with approval decision deadlines before the end of the year.
“We could easily see 50 new drugs approved this year,” said Loss, which would rank 2020 as one of the most productive years ever for new medicines reaching the market. The high-water mark was set in 2018 with 59 drug approvals. Last year, the number was 48.
Gilead’s filgotinib and Biomarin’s Roctavian were widely expected to secure marketing clearance this week, so the rejections were most definitely a surprise. The turndowns came within 12 hours of each other on Tuesday night and Wednesday morning, respectively, which amplified the drama.
But in hindsight, both drugs had issues which should have tempered expectations for approval. Gilead and Biomarin, in their own ways, gambled on flawed drug applications. Blame them, not the FDA, for the consequences.
In an interview with Biopharma Dive on Wednesday, Biomarin CEO Jean-Jacques Bienaime accused the FDA of “moving the goalpost” on the approval requirements for Roctavian, a gene therapy targeting hemophilia A. “We had an agreement with the FDA over one year ago,” he said.
A similar situation happened in June, when the FDA rejected a NASH drug from Intercept Pharmaceuticals. Like Bienaime, Intercept CEO Mark Pruzanski blamed the FDA for blindsiding the company with concerns about its drug at the last moment.
Both Biomarin and Intercept might have legitimate complaints, or not. The FDA, by law and practice, doesn’t talk publicly about rejected drugs. The agency sends so-called Complete Response Letters to companies that explain the reasons for a drug’s rejection, but those letters are confidential. Companies like Biomarin or Intercept could disclose the full content of Complete Response Letters, if they choose, but they didn’t. It’s rarely done.
Instead of worrying about shifts in the FDA’s attitudes towards new drug approvals, investors should demand more transparency from companies when it comes to FDA interactions. Complete Response Letters, with reasonable redactions to protect certain trade secrets, should be made public. Once that happens, there will be fewer surprise FDA drug rejections.