Credit: US Food and Drug Administration

The year 2023 has been unique for the medical community and the US Food and Drug Administration. With the past 3 years dominated by conversations around infectious disease and emergency use authorizations, 2023 has been a return to normalcy, with the agency bringing forth a wide breadth of regulatory decisions, including more than 2 dozen novel drug approvals and landmark decisions for conditions without prior treatments.

To celebrate the achievement, ingenuity, and advancement of the medical field on display thus far in 2023, the HCPLive editorial team has compiled a list of the top FDA approvals of 2023 so far, with our list incorporating coverage from within the HCPLive Network, including NeurologyLive, ContagionLive, and Contemporary OBGYN.

Top Approvals through June 2023

FDA Grants Traditional Approval to Lecanemab as Therapy for Early-Stage Alzheimer Disease

On January 1, 2023, the FDA opened the year by granting Accelerated Approval to lecanemab (Leqembi) treat adult patients with Alzheimer’s Disease. The approval represents the second for a new category of medications approved for Alzheimer’s disease that target the fundamental pathophysiology of the disease.

On July 6, 2023, the FDA expanded the Accelerated Approval of lecanemab to a traditional approval for early-stage Alzheimer disease. The approval of Eisai’s amyloid beta-directed antibody is based on data from the phase 3 CLARITY AD trial.
Related: Lecanemab’s Impact on Care and Diagnosis and the Future of Alzheimer Disease Treatment

FDA Approves Pegcetacoplan Injection for Geographic Atrophy

On February 17, 2023, the FDA approved pegcetacoplan injection (Syfovre) for the treatment of geographic atrophy (GA) secondary to age-related macular degeneration (AMD). The approval, which was granted to Apellis Pharmaceuticals, marks the first and only FDA-approved treatment for GA.

The approval is based on data from the phase 3 OAKS and DERBY trials. In these trials, pegcetacoplan injection reduced the rate of GA lesion growth compared to sham and demonstrated increased treatment effects over time, with results indicating up to a 36% reduction in lesion growth with monthly treatment between months 18-24.
Related: Significance of the Pegcetacoplan Approval for GA, with Charles Wykoff, MD, PhD

FDA Approves Narcan Nasal Spray for Over-the-Counter Use

In the last decade, the word Narcan has become synonymous with the opioid epidemic since its approval as a prescription drug in 2015. Since approval, many have argued greater access could further reduce deaths from the opioid epidemic. This all changed on March 29, 2023, with the FDA’s announcing approval of naloxone hydrochloride nasal spray (Narcan) for nonprescription, over-the-counter use.

For the approval, which applies only to 4mg Narcan nasal spray from Emergent BioSolutions, the company was required to provide data demonstrating the drug’s safety and efficacy as directed in its proposed labeling and show consumers can understand how to use the drug without the supervision of a healthcare professional.

FDA Approves Vowst, the First Oral Live Microbiota Treatment for rCDI

On April 26, 2023, the FDA announced the approval of Vowst for the treatment of the prevention of recurrence of Clostridioides difficile (C. difficile) infection (CDI) in individuals 18 years of age and older, following antibacterial treatment for recurrent CDI. The approval, which comes after the application was granted Priority Review, Breakthrough Therapy and Orphan designations, was awarded to Seres Therapeutics Inc and represents the FDA’s first approval of an oral fecal microbiota product.

Administered through 4 capsules taken once a day, orally, for 3 consecutive days, the agent’s approval is based on a randomized, placebo-controlled ECOSPOR III trial of 182 adult patients with CDI, which concluded use of Vowst was associated with a lower rate of CDI recurrence at 8 weeks than placebo therapy (12.4% vs 39.8%, respectively).
Related: Reaction to the Approval of Vowst for rCDI, with Sahil Khanna, MBBS

FDA Approves GSK’s Arexvy as the World’s First RSV Vaccine for Older Adults

On May 3, 2023, the FDA approved RSVPreF3 +AS01E (Arexvy) for the prevention of lower respiratory tract disease caused by respiratory syncytial virus (RSV) in individuals 60 years of age and older. The approval represents the first RSV vaccine approved in the US and comes after a March 1, 2023, vote from the FDA’s Vaccines and Related Biological Products Advisory Committee in favor of the agent.

The approval is based on safety and efficacy data from an FDA analysis of an ongoing, randomized, placebo-controlled trial conducted in individuals aged 60 years and older. The FDA pointed out it is requiring the company to conduct a postmarketing study to assess the signals of serious risks for Guillain-Barré syndrome and acute disseminated encephalomyelitis.

FDA Approves Fezolinetant for Vasomotor Symptoms

On May 12, 2023, the FDA approved their 16th novel agent for the year with the approval of fezolinetant (Veozah) for the treatment of moderate to severe vasomotor symptoms, or hot flashes, caused by menopause. The agent, which works by binding to and blocking the activities of the NK3 receptor, represents the first NK3 receptor antagonist to receive approval from the FDA to treat moderate to severe hot flashes from menopause.

The approval of fezolinetant is based on the data from the phase 3 BRIGHT SKY Program, which included multiple trials. In these trials, results provided evidence demonstrating the safety and efficacy of the fezolinetant for vasomotor menopausal symptoms as well as providing evidence of the long-term safety of the agent.

FDA Approves B-VEC Topical Gene Therapy for Dystrophic Epidermolysis Bullosa

On May 19, 2023, Krystal Biotech announced the FDA had approved beremagene geperpavec-svdt (Vyjuvek) for the treatment of patients six months of age or older with dystrophic epidermolysis bullosa (DEB). With approval, beremagene geperpavec-svdt became first-ever redosable gene therapy and the first and only medicine approved by the FDA for the treatment of DEB, both recessive and dominant, that can be administered by a healthcare professional in either a healthcare professional setting or in the home.

Approval of the agent is support by data from the phase 3 GEM-3 trial. In the trial, which included 31 patients with DEB, results suggested complete wound healing occurred in 67% of the wounds treated with beremagene geperpavec-svdt as compared with 22% of those treated with placebo at 6 months, with further analysis demonstrating complete wound healing at 3 month was achieved by 71% in the beremagene geperpavec-svdt group relative to 20% of the placebo group.
Related: FDA Approval of B-VEC Gene Therapy for DEB, with Peter Marinkovich, MD

FDA Approves Sotagliflozin for Treatment of Heart Failure

On May 26, 2023, the FDA approved their 22nd novel agent for the year with the approval of Sotagliflozin (Inpefa) for the treatment of heart failure across the full range of left ventricular ejection fraction. An SGLT1/2 inhibitor from Lexicon Pharmaceuticals, the agent becomes the third agent boasting SGLT2 inhibition as a mechanism of action to receive such an indication in heart failure.

The approval of sotagliflozin is based on the data from the SCORED and SOLOIST-WHF trials. In these trials, which were stopped early due to a loss of funding during the COVID-19 pandemic, demonstrated efficacy and safety of the agent in patient populations with type 2 diabetes and chronic kidney as well as those with type 2 diabetes who had been hospitalized for worsening heart failure.
Related: Exploring the Impact of the Sotagliflozin Approval, with Deepak Bhatt, MD, MPH

FDA Approves Sarepta's Landmark DMD Gene Therapy Elevidys

On June 22, 2023, the FDA approved delandistrogene moxeparvovec-rokl (Elevidys) for treatment of pediatric patients 4 through 5 years of age with Duchenne muscular dystrophy (DMD) with a confirmed mutation in the DMD gene who do not have a pre-existing medical reason preventing treatment with this therapy. The approval of delandistrogene moxeparvovec-rokl, which is administered as a single intravenous dose, represents the FDA’s first gene therapy for treatment of certain patients with DMD and was approved through the agency’s Accelerated Approval pathway.
Related: SRP-9001 Approved As First Gene Therapy for Duchenne Muscular Dystrophy