New FDA COVID-19 policy supports vaccines for high-risk groups, clinical trials for others more

When Covid-19 vaccines are launched during the fall and winter respiratory disease seasons, they will continue to be based on the same lineage that is the basis for vaccines in past seasons, but have been adjusted to address variants currently prevalent in the United States and elsewhere in the world.

Last week, the FDA’s advisory committee voted to recommend a unit-price vaccine through the JN.1 lineage. The FDA does not have to follow the committee’s vote, but does so often. In this case, the agency follows the committee, but is to provide vaccine manufacturers with other directions that their vaccine should “prioritize” the use of the LP.8.1 strain, a descendant of JN.1. According to the Centers for Disease Control and Prevention Data Center, LP.8.1 is currently the most extensively circulating variant in the United States.

As agencies change their policy of vaccination, FDA directives for the next season of Covid-19 vaccine are coming. In an article published last week, FDA Commissioner Martin Makary, director of the FDA Center for Biosystem Assessment and Research, Vinay Prasad, director of the FDA Center for Biosystem Assessment and Research, said the agency will highlight the high risk of vaccination for certain populations, such as the elderly, such as facing the serious phenomenon of COVID-19 Infection. For younger, healthier people, it will need to view data from randomized, controlled clinical trials before approving a biolicensing application, the FDA said.

The FDA marks a change in its thinking when approving Novavax’s Covid-19 vaccine. The regulatory decision announced last week only allows vaccines for those aged 65 and older and older, as well as those aged 12 to 64, which puts them at high risk of severe results from Covid-19 infection. This is contrary to the Messenger RNA Covid-19 vaccines sold by Pfizer and ModernA, which have been approved for more widespread use.

The narrower approval of the Novavax vaccine is consistent with discussions last month by the CDC Advisory Committee on Immunization Practice (ACIP). During the ACIP meeting in April, committee members discussed the proposal to change its recommendations to focus on high-risk groups. Makary and Prasad said in the NEJM article that the benefits of repeated administration, especially in low-risk individuals, are uncertain. The FDA will look for the evidence.

“The FDA will approve vaccines for high-risk populations while requiring strong gold-standard data for those at low risk,” Makary and Prasad wrote. “These clinical trials will provide future instructions for the FDA, but more importantly, they will provide information that urgently needs health care providers and the American people.

There is a lot of other news from the FDA. Here is a review of recent regulatory developments:

Regulatory decision-making

– The liquid has received long-awaited FDA approval for Yutrepia, a treatment for pulmonary and pulmonary hypertension associated with interstitial lung disease. The drug is formulated as an inhaled powder administered by a palm-sized device. The active ingredient in Yutrepia is Treprostinil, which is now a universal drug. The new Liquidia drug will compete with the Treprostinil product line sold in combination with therapeutic agents.

– Blocky gsk biopharmaceutical nuclear NUCARA adds chronic obstructive pulmonary disease to its label, the fourth indication. Drug treatment of type 2 inflammation associated with COPD. In this direction, it competes with Type 2 Anti-inflammatory SANOFI DUBIXENT.

– Fugilbio Diagnosis is now the first FDA-based FDA-based blood test for Alzheimer’s disease. Diagnostic tests at the two protein levels are associated with the presence of amyloid plaques in the brain characterized by neurodegenerative disease. Fujirebio has sold a diagnosis of Alzheimer’s, a diagnostic method that tests these proteins in cerebrospinal fluid, which requires an invasive spinal faucet to purchase samples.

– Merck Drig Welireg has expanded its approval to include treatment for children 12 years and older with pheochromocytoma or paraganglioma (PPGL), two rare tumor types. Regulatory decisions make Welireg the only PPGL therapy in the United States. Welireg won the initial approval in 2021 to treat Von Hippel-Lindau disease, a rare genetic disease that causes benign tumors to become cancerous. Merck reported Welireg sales in 2024 were $509 million, up from $218 million the previous year.

– Incyte Drug Retifanlimab has received long-awaited approval for chemotherapy as an advanced treatment for anal canal squamous cell carcinoma. Regulatory decisions also cover the use of the drug as a single drug for this type of cancer, with the development or intolerance of the disease in chemotherapy. The FDA rejected the antibody drug in such a 2021 direction, asking the company to provide more data. Brand Zynyz brand Retifanlimab received its first FDA approval in 2023 to treat metastatic Merkel cell carcinoma. According to Incyte’s annual report, Zynyz made $3.2 million in revenue last year.

– Abbvie’s Emrelis has received accelerated approval from the FDA for the treatment of non-small cell carcinoma characterized by overexpression of a protein called C-MET. The drug is an antibody drug conjugate designed to target the protein. Emrelis represents its first in-house developed solid tumor drug and also represents the first solid tumor FDA approval in lung cancer, Abbvie said.

– Verastem Oncology’s combination therapy is now the first rare type of ovarian cancer therapy approved by the FDA for low-grade serous ovarian cancer (LGSOC). Treatment pairs two oral small molecules, each with a different dosing schedule. Joint processing will be sold by Avmapki Fakzynja.

– European Commission grants marketing authorization to Leqembi for Elzheimer’s disease Patients carrying the APOE4 gene are at greater risk for Alzheimer’s patients and they are at greater risk for complications of Leqembi. For vectors with two copies of the gene, the risk is greater. The committee approved Leqembi only for patients with copy of APOE4.

– Johnson & Johnson Drug Imaavy has received FDA approval for the extension of treating rare neuromuscular disorders, but has a broader label, but with a broader label compared to Argenx and UCB competitors. This is the first approval of Imaavy, the J&J project can earn $5 billion in peak sales in a variety of immune diseases. Imaavy’s $6.5 billion acquisition of Momenta Pharmaceuticals from J&J.

– Akso combined with standard chemotherapy has obtained FDA-approved PENPULIMAB as the first-line treatment for recurrent or metastatic non-metastatic nasopharyngeal carcinoma (NPC) in adults. The agency also approved a Hong Kong-based biotech drug as a monotherapy for adults with metastatic non-rotating NPCs, which has been progressed after or after chemotherapy. Penpulimab, an antibody designed to block the checkpoint protein PD-1, is Akeso’s first in-house developed drug that can be approved by FDA.

– The European Commission has approved AstraZeneca’s susceptibility, as well as standard chemotherapy for care, as a first-line treatment for mantle cell lymphoma (MCL). Oral small molecules are inhibitors of BTK, an enzyme that supports cancer growth. The FDA approved Calquence as a first-line MCL treatment in January. The pills twice daily were initially approved for second-line MCL treatment in 2017.

– FDA has canceled its clinical control of Atara Biothapeutics immunotherapy EBVALLO, a potential treatment for lymphocytic proliferative disease (EBV+LPV) after advanced Epstein Barr virus-positive transplantation. Due to problems with third-party manufacturers, the FDA kept the drug a few days after refusing treatment in January. Ebvallo, a ready-made therapy for bioengineered T cells harvested by healthy donors, won European recognition in EBV+LPV in 2022.

– Zevaskyn of Abeona Therapeutics is a cellular gene therapy made from patients’ own skin cells, obtained FDA approval for the treatment of recessive dystrophic epidermal solute Bullosa, A hereditary disease, lacking the key proteins that hold the skin layers together makes patients susceptible to injury and blisters. Zevaskyn is the third treatment for the FDA-approved epidermal solution in the past two years, adding Vyjuvek from Krystal Biotech and Filsuvez from Chiesi Group.

– Blockbuster Sanofi and Regeneron Pharmaceuticals Dupixent received FDA approval for their seventh instruction: chronic spontaneous urticaria. The FDA rejected Sanofi’s dupixent application in this 2023 direction. The latest regulatory decision for the product is based on a resubmission, which includes data from another 3-phase study.

– Click Therapeutics Mobile App CT-132 to obtain FDA marketing authorization as a treatment for preventing episode migraines. The software joins the Click Digital Therapeutics portfolio, which includes commercial applications for smoking cessation, major depression and type 2 diabetes.

Stumbling and frustration

– The FDA has expanded the regulatory decision-making target date for Biohaven’s Troriluzole in vertebrate ataxia, a rare neurodegenerative disease with no approved treatment. Biohaven expects a decision in the third quarter of this year, but Biotech says the FDA extends the review by three months to give time to review other information the company requested to submit. The FDA also said it plans to convene an advisory committee to discuss Troriluzole.

Troriluzole has a history of clinical trial and regulatory setbacks, including refusal to submit letters in 2023. But discussions with the FDA have led to a new clinical trial protocol that incorporates real-world data. Regulatory submissions are based on positive results from the study reported last year.

– Valneva’s Chikungunya vaccine faces greater regulatory scrutiny due to reports of serious adverse events in older adults, including neurological and cardiovascular complications. The FDA and the Centers for Disease Control and Prevention recommend the administration of the vaccine Ixchiq in adults aged 60 and older. In addition, the European Medicines Agency conducted its own inquiry and said that the vaccine is not allowed to be used in adults aged 65 and above. Valneva said it has “actively participated” with health authorities in all regions of IXCHIQ.

– Global Technology must wait longer to make a decision on its obstructive hypertrophic cardiovirus candidate Aficamten. The agency requires biotech submissions to submit risk assessment and mitigation strategies not included in the original submission. The application’s major amendment extends the drug review by three months; the new target date for regulatory decisions is December 26.

– Health care biotherapies are also facing delays. FDA misses its April 29 target date to determine Elamipretide, a drug that could be the first approved treatment for Barth syndromean ultra-rare mitochondrial disease. The FDA has not given Invisible a new target date to decide. Here is more information about elamipretide and its long-term development and regulatory path.

– FDA is placed in clinical holdings in the Phase 1B test of Vyne Therapeutics’Vyn202, an experimental treatment for moderate to severe plaque psoriasis. Bridgewater, N.J.-based, said the holder recently observed testicular toxicity in dogs in a non-clinical toxicology study. Screening, admission and dose have been suspended in humans. Wien said no serious adverse events were reported in clinical trials.

More changes from the FDA

– The FDA is using AI internally within all centers of the agency with the aim of completing this integration by the end of June. This move is to complete the newly generated AI pilot for the scientific reviewer. According to the FDA, these generated AI tools will allow employees to spend less time on tedious, repetitive tasks that often slow down the audit process. AI is also a key component of the FDA’s plan to phase out drug submission requirements for animal testing. The transition includes accepting data generated by companies using computing technology and AI to predict human drug behavior and side effects.

Illustration: appledesign, Gate Image