- Aimmune Therapeutics expects to know by the end of March whether the Food and Drug Administration will proceed with review of its peanut allergy treatment, the company said Thursday, clearing up some lingering confusion regarding the drug's regulatory status.
- Still uncertain, however, is how the FDA would proceed if it accepts Aimmune's Biologics License Application for approval of AR101, as the biotech's drug is called. Aimmune confirmed Thursday the agency does not consider the treatment to be covered by user free agreements that typically govern most drug reviews.
- Aimmune had thought otherwise when it submitted AR101 last December, only to find out the FDA would not review the drug during the 35-day U.S. government shutdown — an unexpected development that had then caught investors and Wall Street analysts off guard.
Aimmune's announcement, which came along with fourth quarter financials, confirmed what many had suspected after the apparent — and unusual — misunderstanding between the company and the FDA last year.
During the shutdown, the FDA continued to review any drugs for which it had received user fees and which were covered by Prescription Drug User Fee Act agreements between industry and the regulator. Having submitted AR101 and paid its user fee on Dec. 21, Aimmune expected its peanut allergy treatment would fall in that category.
The FDA, however, disagreed and determined initially that AR101 was exempt from PDUFA, Aimmune said Thursday, likely due to it being an allergenic product.
"We had always assumed this was going to be a PDUFA filing," said Aimmune CEO Jayson Dallas on a Feb. 28 earnings call.
Dallas added that, since initially discovering the FDA considered AR101 a non-PDUFA product, his company also learned agency's "intent is to declare that any allergenic product is, in fact, exempt from the PDUFA pathway."
Federal funding restored, the agency is now considering whether to accept Aimmune's application. But the timeline of a review, if the FDA proceeds, could stretch to 12 months or longer, rather than the standard 10 months for PDUFA-eligible products.
Aimmune said Thursday it is continuing discussions with the FDA regarding "the apparent lack of precedent for the FDA reviewing a BLA for a PDUFA-exempt product candidate that has Breakthrough Therapy designation," as AR101 does. The biotech hopes that AR101's designation will lead to a speedier review.
"Regardless of AR101's PDUFA status, FDA is due to accept or reject the BLA filing by the end of March," wrote Piper Jaffray analyst Christopher Raymond in a Feb. 28 note. "We expect at that time, FDA will have indicated whether this Breakthrough Therapy designation confers an expedited review process or not."
Data compiled by Credit Suisse, an investment bank, shows past non-PDUFA allergenic products submitted via a BLA were reviewed over the course of 13 months to 15 months — a timeline that would put any potential approval of AR101 into early 2020.
Even if that were the case, Aimmune could still arrive on market before competitor DBV Technologies, which now expects to resubmit by the third quater a Biologics License Application for its Viaskin Peanut product after voluntarily withdrawing it in December.
Both Aimmune and DBV aim to treat peanut allergy via desensitization, delivering a slowly increasing amount of allergen to boost protection. AR101 is administered orally, whereas Viaskin Peanut uses a patch applied to the skin.