FDA Updates Week of March 28, 2022

FDA clears second COVID-19 recall, approves higher dose of Ozempic, Cabenuva for HIV-positive teens, treatment for rare seizure disorders, and oral testosterone replacement. Agency releases comprehensive response letter for treatment for anemia linked to chronic kidney disease, and advisory board votes against drug for ALS.

FDA clears second COVID-19 vaccine recall.

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

The FDA 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 assess 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 press 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.

The FDA approves Cabenuva for adolescents living with HIV.

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

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

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

The FDA approves a higher dose of Ozempic.

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

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

FDA approves Fintepla for hard-to-treat seizure disorders.

The FDA has approved Fintepla (fenfluramine) from UCB 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 aged two years and older in the United States and the European Union.

It is available in the United States as part of a REMS (Risk Evaluation and Mitigation Strategy) program. Fintepla is a serotonergic drug, and there is an association between these therapies and heart disease that affects the valve and pulmonary arterial hypertension. Before starting treatment, patients should undergo an echocardiogram to establish a baseline before starting treatment and to rule out any pre-existing valvular heart disease or pulmonary hypertension. In addition, echocardiogram monitoring should be performed every six months for the first two years and annually thereafter.

The FDA approves Tlando, an oral testosterone substitute.

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

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

The FDA issues a CRL for vadadustat for CKD anemia.

The FDA has issued a comprehensive response letter to Akebia Therapeutic’s New Drug Application for vadadustat, an investigational oral hypoxia-inducible prolyl hydroxylase factor (HIF-PH) inhibitor under review for the treatment anemia due to chronic kidney disease (CKD).

The agency said the data does not support a favorable assessment of the benefits and risks of vadadustat for dialysis and non-dialysis patients. The FDA has expressed safety concerns noting the 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 thrombosis of vascular access in dialysis patients; and the risk of drug- induced liver injury.

The CRL said Akebia could explore ways to potentially demonstrate a favorable benefit-risk assessment through further clinical trials.

FDA advisory committee votes against ALS drug.

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

AMX0035, developed by Amylyx Pharmaceuticals, is designed to target pathways involved in ALS, including the endoplasmic reticulum and mitochondria-dependent neuronal degeneration. It is a fixed-dose oral 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 by apoptosis.

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