The U.S. FDA hit a five-year approval low last year, greenlighting just 37 novel drugs.
But if the agency keeps its current pace, approval numbers seem poised for recovery in 2023. Seven months into the year, the FDA has approved 35 new molecular entities and new therapeutic biological products, plus 10 vaccines, allergenic products, and cell and gene therapies.
Here are a few decisions that stand out from the crowd.
RSV vaccines
A once-heated RSV race has begun to simmer as several preventatives crossed the finish line in 2023.
A highly contagious and commonly found virus, RSV can cause potentially serious respiratory illness. In older adults, an estimated 177,000 hospitalizations and 14,000 deaths annually in the U.S. are attributed to the virus. In kids under five, RSV results in around 58,000-80,000 hospitalizations and 100-300 deaths in the U.S. per year.
Despite this, there were no FDA approved vaccines until this past May when the agency gave the OK to GSK’s Arexvy for use in individuals aged 60 and older. A month later, the agency approved Pfizer’s bivalent RSV vaccine, Abrysvo, for the same patient population.
Most recently, AstraZeneca and Sanofi’s single-dose long-acting monoclonal antibody, branded Beyfortus, became the first U.S. approved RSV preventive option for a broad infant population.
There is more to come as well. Pfizer’s Abrysvo is also vying for approval for use in pregnant mothers,* with a regulatory action date in late August. In July, Moderna initiated the rolling submission process with the FDA for its mRNA-based RSV vaccine for use in older adults.
CGT approvals
Last year, the U.S. saw a record-setting five CGT commercial approvals, including the first gene therapy authorized for hemophilia B. During Phacilitate’s Advanced Therapies Week back in January, 2022 was commemorated as ‘the year the commercial reality of CGT became apparent’ — but 2023 might surpass it. 2023 now has matched 2022 with five approvals classified as CGT by the agency.
The run started in April when the FDA approved Gamida Cell’s Omisirge, an allogeneic cell therapy used for the treatment of blood cancers. In mid-May the agency approved Krystal Biotech’s topical gene therapy for the repeated treatment of a rare skin disease. The drug, branded Vyjuvek, is the first-ever redosable gene therapy and the first and only medicine approved by the FDA for the treatment of dystrophic epidermolysis bullosa.
In June, within a week’s span, the CGT sector notched three more approvals. On June 22, Sarepta Therapeutics announced that the FDA had granted accelerated approval for Elevidys, a one-time gene therapy based on an adeno-associated virus, approved for the treatment of pediatric patients with Duchenne muscular dystrophy.
Days later, on June 28, the agency approved CellTrans’ Lantidra, marking the first allogeneic pancreatic islet cell therapy made from deceased donor pancreatic cells for the treatment of type 1 diabetes. (Some in the industry argue that Lantrida is not a true cell therapy, however the FDA classifies it as such.) The next day, the FDA approved BioMarin Pharmaceutical’s Roctavian, the first and only gene therapy for adults with severe hemophilia A.
Alzheimer’s treatment
In early July, the FDA officially changed the approval status of the Alzheimer’s treatment Leqembi, co-commercialized by Eisai and Biogen, from accelerated approval to traditional approval.
An anti-amyloid beta protofibril antibody, Leqembi initially received approval in January through the accelerated approval pathway, but not without controversy, including two clinical trial deaths linked to the drug being used in combination with blood thinners.
But the FDA was satisfied with confirmatory data presented in a phase 3 study in which Leqembi, which works by reducing amyloid plaques that form in the brain, delayed cognitive decline by 5.3 months after 18 months of treatment.
With the recent approval expansion, Leqembi will now be covered by Medicare for Alzheimer’s patients.
On track
Eli Lilly, whose bid for accelerated approval of its Alzheimer’s drug, donanemab, was rejected by the FDA back in January, has resubmitted the drug for traditional approval. Lilly says it expects regulatory action by the end of the year.
With several more highly-anticipated PDUFA dates on the books — Iovance Biotherapeutics’ lifileucel which has the potential to be the first individualized, one-time cell therapy for patients with advanced melanoma; Vertex and CRISPR Therapeutics’ sickle cell disease therapy, exa-cel, which would be the first CRISPR gene-edited therapy approved by the FDA; and bluebird bio’s lovo-cell, a personalized one-time gene therapy also for the treatment of sickle cell disease — 2023 promises to be a good year for drug innovation.
Updates:
*On August 21, the FDA approved Pfizer's Abrysvo for use in pregnant individuals
