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FDA commish finding his feet, COVID-19 and chaotic communication hitting speedy approvals

The FDA’s accelerated approvals program is on shaky ground after a series of surprise rejections, poor communication and COVID-19-focused staff.

This is according to analysis by an anonymous FDA consultant out of regulatory firm Prevision Policy, speaking to analysts at Cowen, who see the recent spate of surprising complete response letters for BioMarin, Intercept, Gilead/Galapagos and Tricida as mounting evidence that the regulator may be backing off accelerated approvals, at least outside of cancer, where speedy reviews are still the norm, and question whether complete response letters are becoming more likely.

The accelerated approval program from the FDA allowed drugs for serious conditions that filled an unmet medical need to be approved based on a surrogate endpoint, something that speeds up trials but does leave the FDA open to criticism about how well this endpoint points to efficacy.

BioMarin’s rejection for its hemophilia A gene therapy valrox seemed to be a big surprise to the biotech, and it said the FDA’s qualms over durability came out of the blue. “Having previously agreed with the Agency on the extent of data necessary to support the BLA, the FDA introduced a new recommendation for two years of data from the company’s ongoing 270-301 study (phase 3) to provide substantial evidence of a durable effect using Annualized Bleeding Rate (ABR) as the primary endpoint,” the company said in a statement earlier this month.

“The Agency first informed the company of this recommendation in the complete response letter having not raised this at any time during development or review.”

Some of these issues, the Prevision consultant said in a discussion with Cowen, may be down to the virtual FDA meetings being made amid COVID-19, which is also lending to problems with communication and could go someway to explaining what happened with BioMarin’s complete response letter, as well as Tricida’s.

“With all meetings being held virtually, it is understandable that messages are not as clearly communicated as in the past,” the consultant said. “In fact, Tricida’s management suggests that the inability to hold in person meetings played some part in the FDA’s issuance of the CRL to [metabolic acidosis drug] veverimer.”

There is also an issue with FDA personnel. “Accelerated Approval proponent Commissioner Scott Gottlieb left FDA at the start of 2019. His successor, Stephen Hahn, lacked the regulatory experience to engage deeply on issues like AA upon joining the agency, and his agenda has been dominated by the COVID response ever since.

“Center for Drug Evaluation & Research Director Janet Woodcock, a highly effective advocate for the accelerated approval pathway, has also been taken out of the direct line of review oversight, moving to a temporary post in the Commissioner’s office working on COVID response activities.”

It’s not time to panic, the Prevision consultant said, as: “Overall, Prevision thinks that the FDA remains highly functional with a healthy pace of approvals despite the disruptions. However, against that backdrop, the ability of sponsors to use the accelerated approval pathway outside of oncology does bear monitoring.”

This comes against the backdrop of the FDA approving 37 new molecular entities so far this year, a strong number for any year, let alone one with a pandemic.

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