FDA Updates Week of March 28, 2022

The FDA authorizes second COVID-19 booster and approves a higher does of Ozempic, Cabenuva for adolescents with HIV, a therapy for rare seizer disorder, and an oral testosterone replacement. The agency issues a complete response letter for a therapy for anemia related to chronic kidney disease, and an advisor committee votes down a drug for ALS.

FDA authorizes second COVID-19 vaccine booster.

The FDA authorized a second booster dose of either the Pfizer-BioNTech or the Moderna COVID-19 vaccines for people 50 and older and certain immunocompromised individuals.

FDA’s decision applies only to the Pfizer-BioNTech and Moderna COVID-19 vaccines, and the authorization of a single booster dose for other age groups with these vaccines remains unchanged. “The agency will continue to evaluate data and information as it becomes available when considering the potential use of a second booster dose in other age groups,” the FDA said in a news release.

The FDA determined that the benefits of the second booster dose outweighed the risks after reviewing data from Pfizer and BioNTech, Moderna, and sources outside of those companies.

FDA approves Cabenuva for adolescents with HIV.

The FDA has approved Cabenuva (cabotegravir and rilpivirine) for the treatment of HIV-1 in virologically suppressed adolescents who are 12 years of age or older, weigh at least 35 kg and are on a stable antiretroviral regimen, with no history of treatment. Co-developed as part of a collaboration between Janssen and ViiV Healthcare, Cabenuva is the first long-acting HIV-1 treatment regimen, to be made available for eligible adolescents.

Cabenuva is approved as a once-monthly or every-two-months treatment for HIV-1 in virologically suppressed adults and adolescents. It contains ViiV Healthcare’s cabotegravir and Janssen’s rilpivirine in a single-dose vial.

Additionally, the FDA approved a label update that made the oral lead-in period optional for adults living with HIV-1 who planned to begin the injectable treatment regimen. The oral lead-in period is also optional for adolescent patients.

FDA approves higher dose of Ozempic.

The FDA has approved a 2 mg dose of Ozempic (semaglutide) injection, a once-weekly glucagon-like peptide-1 (GLP-1) analog to improve blood sugar in adults with type 2 diabetes. Developed by Novo Nordisk, Ozempic is now available in three doses (0.5 mg, 1 mg, and 2 mg) to help people with type 2 diabetes reach their blood sugar (A1C) goal.

The approval is based on the phase 3 SUSTAIN FORTE, which found the higher dose of Ozempic helped patients who needed additional glycemic. In the trial, people with an average starting A1C of 8.9% treated with Ozempic 2 mg achieved a reduction in blood sugar of 2.1% at week 40 compared with 1.9% with Ozempic 1 mg. With the higher dose, the most common adverse events were gastrointestinal. Gastrointestinal adverse reactions occurred more frequently among patients receiving Ozempic 2 mg (34.0%) vs Ozempic 1 mg (30.8%).

FDA approves Fintepla for difficult-to-treat seizure disorder.

The FDA has approved UCB’s Fintepla (fenfluramine) for the treatment of seizures associated with Lennox-Gastaut syndrome in patients two years of age and older. The agency also granted pediatric exclusivity for the product. Fintepla is already approved for the treatment of seizures associated with Dravet syndrome in patients two years of age and older in the United States and European Union.

It is available in the United States through a Risk Evaluation and Mitigation Strategy (REMS) Program. Fintepla is serotonergic drug, and there is an association between with these therapies and heart disease that impacts the valve and pulmonary arterial hypertension. Prior to starting treatment, patients must undergo an echocardiogram to establish a baseline prior to initiating treatment and exclude any pre-existing valvular heart disease or pulmonary hypertension. Additionally, echocardiogram monitoring should be conducted every six months for the first two years and annually thereafter.

FDA approvals Tlando, an oral testosterone replacement.

The FDA has granted final approval for Tlando (testosterone undecanoate), a treatment for testosterone replacement therapy for conditions associated with a deficiency or absence of endogenous testosterone, or hypogonadism in adult males. Developed by Antares Pharma, Tlando is an oral therapy that does not require dose titration. It had been granted tentative approval as a twice-daily therapy.

Most marketed testosterone replacement therapy products often require multiple dose adjustment clinic visits to achieve the desired testosterone levels. Investigators in a phase 3 trial said a therapy without dose titration has the potential to improve patient compliance. This study, published in the January 2022 issue of Andrology, compared a dosing regimen without titration with with the efficacy and safety obtained from the previous one-year phase 3 study of Tlando with titration.

FDA issues CRL for vadadustat for CKD anemia.

The FDA has issued a complete response letter to Akebia Therapeutic's new drug application for vadadustat, an investigational oral hypoxia-inducible factor prolyl hydroxylase (HIF-PH) inhibitor under review for the treatment of anemia due to chronic kidney disease (CKD).

The agency indicted that the data do not support a favorable benefit-risk assessment of vadadustat for dialysis and non-dialysis patients. The FDA expressed safety concerns noting failure to meet non-inferiority in major adverse cardiovascular events (MACE) in the non-dialysis patient population, the increased risk of thromboembolic events, driven by vascular access thrombosis in dialysis patients, and the risk of drug-induced liver injury.

The CRL stated that Akebia could explore ways to potentially demonstrate a favorable benefit-risk assessment through new clinical trials.

FDA advisory committee votes down ALS drug.

The FDA’s Peripheral and Central Nervous System Drugs Advisory Committee voted 6 to 4 against whether the data from the single trial for AMX0035 establishes it as an effective treatment of amyotrophic lateral sclerosis (ALS). At issue was whether a single phase 2 trial was enough to demonstrate the drug slowed the progression of the disease.

AMX0035, developed by Amylyx Pharmaceuticals, is designed to target the pathways in involved in ALS, including the endoplasmic reticulum and mitochondrial-dependent neuronal degeneration. It is oral fixed-dose combination of two small molecules: sodium phenylbutyrate (PB), which is a small molecular chaperone designed to reduce the unfolded protein response, preventing cell death, and taurursodiol (TURSO; also known as ursodoxicoltaurine), which is a Bax inhibitor designed to reduce cell death through apoptosis.