FDA decisions to watch in 2023

March 29, 2023
The agency is facing a busy year, with potential novel drug approvals ranging from gene therapies to RSV jabs

The U.S. Food and Drug Administration is gearing up for a busy 2023, with novel drug approval hopefuls ranging from gene therapies to RSV jabs. Here is a look at upcoming agency decisions to keep an eye on. 

Perrigo’s OTC birth control 

When the United States Supreme Court overturned Roe v. Wade last year, pharma suddenly found itself in uncharted, highly explosive territory. As state-level abortion restrictions continue to change, companies are searching for ways to ensure market opportunity for their reproductive health drugs.  

This year, the FDA will meet to decide whether or not to approve what would be the country’s first birth control pill available without a prescription. The pill, made by Perrigo, is a progestin-only daily birth control pill — also referred to as a mini pill or non-estrogen pill — branded as Opill. Opill was first marketed by Pfizer, initially approved by the FDA in 1973. HRA acquired it in 2014 but has not been sold in the U.S. in over a decade.  

 Perrigo's HRA Pharma applied for an Rx-to-OTC switch for Opill back in July of last year. Then, in October, a month before a planned FDA advisory committee meeting, the agency postponed the meeting, as well as delayed the drug's PDUFA date for 90 days. Perrigo recently revealed that the rescheduled AdComm meeting will take place this coming May 9-10.

Update: On May 10, 2023, Perrigo announced that an FDA advisory panel voted 17 to 0 with no abstentions that the benefits of making Opill available for over-the-counter use outweigh the risks.

Update: On July 13, 2023, the FDA approved Perrigo's Opill as the first OTC birth control available in the U.S. 

RSV jab(s) 

As COVID vaccines move out of the spotlight, the race for a respiratory syncytial virus (RSV) vaccine continues to intensify with pharma juggernauts like Pfizer, GSK and Sanofi all neck and neck chasing the first approval.  

In November 2022, GSK received Priority Review for its RSV vaccine candidate in older adults — giving the drugmaker a leg up with a regulatory action date of May 3, 2023. GSK’s jab was granted its designation based on results from the AReSVi-006 phase 3 trial, which showed high overall vaccine efficacy against RSV lower respiratory tract disease in adults aged 60 years and older. In early March, an FDA advisory panel voted unanimously 12-0 on the vaccine's effectiveness and 10-2 on safety, teeing the candidate up for approval.

Back in December, Pfizer announced that it had received Priority Review for its vax candidate, RSVpreF, which had also been granted Breakthrough Therapy designation earlier last year. The designation shortened the review period by four months, granting RSVpreF a PDUFA date this coming May.

The vaccine, which uses crystal structure of prefusion F technology to target the viral fusion protein (F) that the virus uses to attack human cells, is being study for use in both older adults and pregnant women. In a phase 3 trial of 37,000 older adults, the vaccine showed 85.7% efficacy in reducing the risk of severe lower respiratory tract illness.

Nirsevimab, jointly developed by Sanofi and AstraZeneca, is designed for the prevention of RSV in newborns and infants entering or during their first RSV season and for children up to 24 months of age who remain vulnerable to severe RSV disease.

The FDA accepted the partners' BLA back in January. If approved, the single-dose vaccine would be the first protective option against RSV disease for all infants. The jab has a PDUFA date in the third quarter of 2023.

Update: On May 19, 2023, an FDA Advisory Committee voted to back Pfizer’s RSV vaccine candidate, Abrysvo, for infants younger than 6 months when administered during the second or third trimester of pregnancy in maternal patients. 

Roche-Sarepta DMD gene therapy  

When Roche bought Sarepta in 2019 for $1.15 billion, SRP-9001 was a driving force behind the acquisition. In another potential landmark approval this year, the agency will decide the fate of the revolutionary treatment for Duchenne muscular dystrophy. Because Duchenne is caused by a mutation in the Dystrophin gene, SRP-9001 is designed to introduce a functional copy of a shortened gene that codes for that protein directly to the muscle tissue. 

The agency accepted Sarepta’s BLA back in November, granting it Priority Review and assigning a PDUFA date of May 29, 2023. 

A few weeks ago, the agency reversed course, deciding it needed to hold an AdComm meeting after all, which presumably will take place prior to the PDUFA deadline.

Per the deal with Roche, Sarepta will be responsible for global development and manufacturing upon receiving FDA approval. Ahead of the approval, Sarepta signed a manufacturing deal with Catalent to manufacture the drug. Their agreement also helped structure how the CDMO may support multiple gene therapy candidates in Sarepta’s pipeline.  

Update: On May 15, 2023, the FDA’s Cellular, Tissue and Gene Therapies Advisory Committee narrowly voted to back its approval

AbbVie-Genmab bispecific antibody for lymphomas 

The result of a broad oncology collaboration between Genmab and AbbVie, epcoritamab's Biologics Licensing Application was accepted with Priority Review designation last November.

Epcoritamab is the result of a partnership that started in 2020, in which AbbVie paid Genmab $750 million with potential milestone payments totaling $2.15 billion. Using AbbVie's payload and ADC technology, the two have worked together to select and develop differentiated next-generation antibody-based product candidates. 

The drug is an anti-CD20 bispecific for lymphomas, designed to simultaneously bind to CD3 on T cells and CD20 on B-cells and induces T cell-mediated killing of lymphoma B cells. In trials, patients treated with the drug were found to have a confirmed overall response rate of 63.1% by an independent review committee.  

Epcoritamab’s PDUFA date is set for May 21, 2023. Industry analysts have forecasted peak sales up to $2.75 billion for the drug.  

Update: On May 19th, 2023, the FDA approved epcoritamab as the first and only T-cell engaging bispecific antibody for the treatment of adult patients with relapsed or refractory (R/R) diffuse large B-cell lymphoma (DLBCL). 

Astellas menopause drug  

Astellas first got its hands on fezolinetant in 2017, when it completed the acquisition of Ogeda for $500 million upfront. Now a potential blockbuster, the treatment is projected to reach an annual total of $1.21 billion in U.S. sales by 2032, according to GlobalData's Expiry Model.  

The Tokyo-based drugmaker's NDA for the selective neurokinin 3 receptor (NK3R) antagonist was accepted back in August 2022. The application hinged on three phase 3 trials which enrolled 2,800 women from across the U.S., Canada and Europe. Last month, the FDA pushed back the drug's regulatory action date to May 22, 2023. 

If approved, the drug would offer a non-hormonal treatment for moderate to severe vasomotor symptoms associated with menopause, such as hot flashes and night sweats.  

Update: On May 15th, 2023, the FDA  approved Astellas Pharma's once daily pill for the treatment of menopause symptoms.

Biogen-Sage depression treatment  

Co-developed by Biogen and Sage Therapeutics, zuranolone, a neuroactive steroid, is being evaluated as a 14-day, rapid-acting, once-daily, oral treatment in adults with major depressive disorder (MDD) and postpartum depression (PPD) 

If approved, zuranolone would be the first oral medication specifically indicated to treat PPD, one of the most common medical complications during and after pregnancy, affecting approximately one in eight women who have given birth in the U.S. 

Zuranolone has a novel mechanism of action as a positive allosteric modulator of GABA-A receptors. It's designed to work by rapidly rebalancing dysregulated neuronal networks to help reset brain function.

The application has been granted Priority Review and the FDA has assigned a PDUFA action date of August 5, 2023. 

Eli Lilly ulcerative colitis drug

Mirikizumab, an anti-IL-23 mAb in development for the treatment of ulcerative colitis, is expected to join the biologics blockbusters club if approved, with projected sales reaching a total of $14.6 billion by the end of 2038.

The drug, originally developed for psoriasis, achieved statistically superior rates of clinical remission at 12 weeks compared to patients taking placebo in phase 3 trials in ulcerative colitis. It currently has a regulatory action date in May of this year. 

If approved, mirikizumab would become the first and only anti-IL23p19 treatment for people with UC. 

Update: On April 14, 2023, the FDA issued a complete response letter for mirikizumab's  BLA, citing issues related to the proposed manufacturing of the drug, with no concerns about the clinical data package, safety, or label for the medicine.

About the Author

Andrea Corona | Senior Editor

Andrea Corona serves as the Senior Editor of Pharma Manufacturing — a leading source of news and insights for pharma professionals — and is responsible for creation of editorial content, moderating webinars, and co-hosting the "Off script" podcast. Her editorial journey started as an as associate editor at Biocompare, an online platform providing product information, industry news, articles, and other resources to support scientists in their work. Before Biocompare, she was a digital producer at Science Friday, focusing on adapting radio segments for the web and social media management. Andrea earned her bachelor's degree in journalism and biology from the State University of New York, at Purchase College.