Since 21 October, the 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, the 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 reviewand 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, the FDA granted accelerated approvalto BeiGenes 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, the FDA granted an accelerated approvalto 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, butwon approval on 20 November. But other recent approvals, like SK Life Sciences Xcopri (cenobamate tablets) to treat partial-onset seizures in adults, and Shionogis 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 formoving too quickly. An article inJAMA Internal Medicinelast summer found that few cancer drugs approved via the accelerated approval pathway improved survival in confirmatory trials.

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

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

Janet Woodcock, director of the FDAs Center for Drug Evaluation and Research, explained recentlythat 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 willcost$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 willcost$575,000 per year.

RAPS: First published in Regulatory Focus by the Regulatory Affairs Professionals Society, the largest global organization of and for those involved with the regulation of healthcare products. Click here for more information.

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FDA approves 5 new costly drugs well ahead of PDUFA dates - Endpoints News

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