Regulatory Focus™ > News Articles > 2019 > 11 > FDA Approves 5 New Costly Drugs Well Ahead of PDUFA Dates

FDA Approves 5 New Costly Drugs Well Ahead of PDUFA Dates

Posted 26 November 2019 | By Zachary Brennan 

FDA Approves 5 New Costly Drugs Well Ahead of PDUFA Dates

Since 21 October, the US Food and Drug Administration (FDA) has been on a tear in approving five new drugs (all with list prices of more than $100,000 per year) months ahead of when they were expected to be approved.

For instance, FDA signed off on Vertex Pharmaceuticals’ Trikafta (elexacaftor/ivacaftor/tezacaftor), a new treatment for those with the most common cystic fibrosis mutation, after only three months of review and well ahead of its 19 March 2020 user fee action date.

On 14 November, more than three months ahead of its 27 February 2020 action date, FDA granted accelerated approval to BeiGene’s Brukinsa (zanubrutinib) for the treatment of patients with mantle cell lymphoma who have received at least one prior therapy.

One day later, Novartis’ Adakveo (crizanlizumab-tmca) won approval for its sickle cell disease treatment two months ahead of its PDUFA date in mid-January 2020. And yesterday, FDA granted an accelerated approval to another sickle cell drug, Global Blood Therapeutics’ Oxbryta (voxelotor), three months ahead of its PDUFA date.

Alnylam Pharmaceuticals’ Givlaari (givosiran), meanwhile, had a PDUFA date of 4 February 2020, but won approval on 20 November. But other recent approvals, like SK Life Sciences’ Xcopri (cenobamate tablets) to treat partial-onset seizures in adults, and Shionogi’s complicated urinary tract infection drug Fetroja (cefiderocol), won approvals near their PDUFA dates.

The string of quick approvals may provide more ammunition for those who criticize the agency for moving too quickly. An article in JAMA Internal Medicine last summer found that few cancer drugs approved via the accelerated approval pathway improved survival in confirmatory trials.

However, viewers of the recent Senate committee hearing considering a new FDA commissioner have seen there are still senators who believe FDA is not moving quickly enough with some approvals.

As the proportion of new drugs receiving expedited approvals in recent years has been increasing, so has the number of approvals for rare diseases.

Janet Woodcock, director of FDA’s Center for Drug Evaluation and Research, explained recently that the agency is working on its own analyses to provide “a more robust response” to these critiques of its approval standards.

She also explained how the high number of approvals in recent years for rare diseases may be influencing this perception of a lower bar, especially as more treatments are approved on the basis of a single-arm study or with an external control group. In addition, she pointed to the “astoundingly” high launch prices for some of these rare disease treatments that may also be part of the reason for the pushback.

Indeed, before discounts, Trikafta will cost $311,503 annually, Brukinsa will cost $12,935 for a 30-day supply, Adakveo will cost between $7,000 and $9,500 per month ($84,000 to $114,000 per year), Oxbryta will cost $125,000 per year and Givlaari will cost $575,000 per year. 

Regulatory Focus newsletters

All the biggest regulatory news and happenings.

Subscribe