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FDA Updates for the Week of Dec. 5, 2022


In COVID-19 news, the FDA has expanded the updated bivalent vaccines EUA to children. The FDA has requested athe removal of Pepaxto indication for multiple myeloma. Regulators have also accepted several applications for: Biogen’s Actemra biosimilar, Pfizer’s RSV vaccine, a resubmitted BLA for Fabry disease. Two companies — Janssen’s and Alnylam — have submitted new applications.

FDA expands updated COVID-19 vaccines EUA to children.

The FDA amended the emergency use authorizations (EUAs) of the updated (bivalent) Moderna and Pfizer-BioNTech COVID-19 vaccines to include use in children starting at six months old.

The decision means that children six months through five years of age who received the original (monovalent) Moderna COVID-19 vaccine are now eligible to receive a single booster of the updated (bivalent) Moderna COVID-19 vaccine two months after completing a primary series with the monovalent Moderna COVID-19 vaccine, FDA said in a news release.

In mid-October, the FDA issued an EUA for both the Moderna and Pfizer updated COVID-19 vaccine boosters in children. These bivalent booster vaccines add the omicron variant BA.4 and BA.5 to the original SARS-CoV-2 and a component of omicron lineage BA.1. Moderna’s updated booster is authorized for children six years of age and older. In August, the FDA had authorized Moderna’s updated booster for those 18 years and older.

FDA requests removal of Pepaxto indication for multiple myeloma.

The FDA has requested that Oncopeptides voluntarily withdraw the indication for Pepaxto (melphalan flufenamide, also called melflufen). The request is based on the outcome of the confirmatory phase 3 OCEAN study, which demonstrated an overall survival hazard ratio of 1.1 in the intent to treat population. A hazard ratio of more than 1 indicates a risk of harm.

But a company spokesperson said that Oncopeptides disagrees with the agency´s assessment of the data from the OCEAN study and has not withdrawn the U.S. indication. “We will carefully consider what is the best option for the patients and the company. We expect a decision to be made before end of Q1, 2023,” the spokesperson said.

In a press release, the company said Pepaxto demonstrated significant survival differences for both melflufen and the comparator drug pomalidomide for large relevant patient groups. The therapy had been granted accelerated approval in the United States on Feb. 26, 2021, to be used in combination with dexamethasone to treat adult patients with relapsed or refractory multiple myeloma.

FDA accepts Biogen’s application for Actemra biosimilar.

he FDA has accepted for review the abbreviated biologics license application (aBLA) for BIIB800, a biosimilar referencing Genentech’s Actemra (tocilizumab), an anti-interleukin-6 receptor monoclonal antibody. Actemra is indicated for several indications, including moderate-to-severe rheumatoid arthritis in adults as well as juvenile idiopathic polyarthritis and systemic juvenile idiopathic arthritis. In 2021, Roche’s U.S. sales of Actemra totaled $1.9 billion

The FDA filing is supported by phase 3 data from a comparative clinical trial demonstrating equivalent efficacy and a comparable safety and immunogenicity profile at week 24 to the reference product. The data were presented at the European Congress of Rheumatology on June 3, 2022.

FDA accepts for priority review BLA for RSV vaccine.

The FDA has accepted for priority review a biologics license application (BLA) for Pfizer’s respiratory syncytial virus (RSV) vaccine, PF-06928316 or RSVpreF, in people 60 years of age and older. The Prescription Drug User Fee Act (PDUFA) goal date for a decision is in May 2023.

RSV is a contagious virus that can cause serious respiratory illness, affecting the lungs and breathing passages. It can be potentially life-threatening for older adults and adults with certain medical conditions. Each year it is estimated that 336,000 older adults are hospitalized globally due to RSV. In the United States, RSV infections in older adults account for about 177,000 hospitalizations and 14,000 deaths each year.

Pfizer’s bivalent vaccine candidate is composed of two preF proteins selected to optimize protection against RSV A and B strains. The vaccine builds on discoveries including those made at the National Institutes of Health (NIH), which detailed the crystal structure of prefusion F, a key form of the viral fusion protein that RSV uses to enter human cells.

FDA accepts resubmitted BLA for Fabry disease.

The FDA has accepted the resubmitted biologics license application for pegunigalsidase alfa to treat adult patients with Fabry disease. Fabry disease is a life-threatening, rare genetic disorder that can cause pain and impaired peripheral sensation and can lead to organ failure, particularly of the kidneys. It is caused by a deficiency of the lysosomal α–Galactosidase–A enzyme, which leads to abnormal deposits of a fatty substance called globotriaosylceramide (Gb3) in blood vessel walls. Fabry disease occurs in one person per 40,000 to 60,000.

Pegunigalsidase alfa (PRX–102) is an enzyme replacement therapy that was developed by Protalix BioTherapeutics and Chiesi Global Rare Diseases. It is a long-acting and chemically modified stabilized version of the recombinant α–Galactosidase–A enzyme. A Prescription Drug User Fee Act (PDUFA) action date is set for May 9, 2023

Janssen submits BLA for another bispecific antibody for multiple myeloma.

Janssen Pharmaceuticals has submitted to the FDA a biologics license application (BLA) for talquetamab to treat patients with relapsed or refractory multiple myeloma. Talquetamab is a bispecific antibody that targets both CD3 receptor on T cells and GPRC5D, which is overexpressed on myeloma cells. Multiple myeloma is an incurable blood cancer that affects plasma cells, which are found in the bone marrow. In 2022, it is estimated that more than 34,000 people will be diagnosed with multiple myeloma, and more than 12,000 people will die from the disease in the United States.

This BLA is supported by data from the phase 1/2, MonumenTAL-1 study of talquetamab in patients with relapsed or refractory multiple myeloma who have received more than three prior lines of therapy. The first presentation of phase 1/ 2 combined results from the MonumenTAL-1 study will be highlighted at the American Society of Hematology (ASH) Annual Meeting on December 10, 2022.

Alnylam submits sNDA for Onpattro in ATTR amyloidosis with cardiomyopathy.

Alnylam Pharmaceuticals has submitted a supplemental new drug application (sNDA) to the FDA for Onpattro (patisiran) to treat patients with transthyretin-mediated (ATTR) amyloidosis with cardiomyopathy. Onpattro is currently approved to treat polyneuropathy of hereditary ATTR amyloidosis in adults.

In cardiomyopathy, the heart loses its ability to pump blood. In ATTR amyloidosis, the ATTR protein can build up in the heart muscle, leading to shortness of breath and swelling in the legs. Patients with ATTR cardiomyopathy can also have arrhythmias that can lead to stroke or death.

The submission is based on positive results from APOLLO-B, a phase 3 global study that demonstrated the effects of patisiran on functional capacity and quality of life in patients with ATTR amyloidosis with cardiomyopathy. The safety profile in APOLLO-B was consistent with what was observed in APOLLO and in postmarketing use of Onpattro, the company said in a press release. In APOLLO-B, the majority of adverse events were mild or moderate in severity.

The 12-month results from the study were presented at the 18th International Symposium on Amyloidosis (ISA) on Sept. 8, 2022.

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