Editors' (re)View: Women's health wins; DMD gene therapy closer to approval

May 19, 2023
Pharma Manufacturing editors Karen Langhauser and Andrea Corona comment on the notable happenings in the pharma industry from the week of May 15

Editor’s note: Welcome to Editors' (re)View, our editors’ takes on things going on in the pharma world that deserve some extra consideration.

It's been a productive few weeks for women's health drugs...

Perrigo recently shared some good news about its OTC birth control pill, revealing that an FDA advisory committee voted unanimously 17-0 in favor of allowing sale of its oral contraceptive without a prescription. If approved, this will be the first OTC birth control pill in the U.S., where unintended pregnancies are hovering around 50%.  

Perrigo's HRA Pharma applied for the Rx-to-OTC switch for Opill back in July of last year. Then, in October, a month before the planned AdComm meeting, the agency postponed, which then delayed the drug's PDUFA date by 90 days. Now, having aced the AdComm, Perrigo will soon have its answer. It does not appear as though the company has shared the exact PDUFA date but it should be late summer.

While it may seem as though this is taking forever, when compared to historical OTC journeys in reproductive health, things are moving at a record pace. The sector’s only example is the emergency contraceptive drug, Plan B, which took ten years to go from prescription to OTC. Susan Wood, who resigned her position as head of the FDA’s Office of Women’s Health because of the Plan B regulatory delays, later referred to the unfoldings as a “tortuous, and indeed politicized, process of approving a safe and effective, but time-sensitive, contraceptive product for over-the-counter sale.”

And, on the other end of the spectrum, last week the FDA approved Astellas Pharma's once daily pill for the treatment of menopause symptoms.

The drug, branded Veozah, offers the first nonhormonal treatment for moderate to severe vasomotor symptoms (a fancy way of saying hot flashes and night sweats) associated with menopause.

Regulators made Astellas sweat out the approval a bit (pun intended). The Tokyo-based drugmaker's NDA was accepted back in August 2022. Astellas cashed in its very valuable (to the tune of $97M) Priority Review voucher to speed up the drug's review, and the drug was assigned a PDUFA date in February. Then, just days before its decision date, the FDA pushed back its decision to May 22, 2023, citing the need for more time to review the drug.

Persistence appears to be key in this space and I'm happy to see it paying off for drugmakers and those who need these drugs.

–Karen Langhauser

Long-awaited DMD gene therapy is backed by FDA advisors 

Earlier this week, we reported that in a narrow 8-6 vote, the FDA’s Cellular, Tissue and Gene Therapies Advisory Committee (CTGTAC) voted to back the approval of Sarepta Therapeutics' gene therapy for Duchenne muscular dystrophy.

Documents released ahead of the meeting stated that the FDA “notes that the clinical studies conducted to date do not provide unambiguous evidence that SRP-9001 is likely beneficial for ambulatory patients with DMD,” adding that the agency had “safety concerns related to the possibility of administering an ineffective gene therapy." And during Friday’s hearing, some committee members reiterated their apprehension regarding the treatment’s usage of engineered viruses for delivery, sharing concern over alternative treatments for patients if Sarepta's therapy were to be deemed ineffective in the future.

The committee's discussion included assessing trial results and the design of the SRP-9001 product itself. Non-clinical evidence and efficacy and safety data from studies 101, 102, and 103, along with an integrated analysis comparing functional outcomes to an external control, further supported SRP-9001.

The BLA for SRP-9001 is undergoing priority review by the FDA, and a regulatory action is expected by May 29, 2023. 

SRP-9001 — a driving force behind Roche’s $1.5 billon acquisition of Sarepta in 2019 — is slated to be manufactured by Catalent should it win approval. After FDA advisors voted to back its approval, Sarepta's shares went up by 25% over the weekend. If approved by the FDA, the drug would be the first gene therapy for DMD patients and is expected to generate a billion dollars in revenue. 

— Andrea Corona