FDA Updates for Week of Aug. 29, 2022

FDA authorizes Pfizer, Moderna updated COVID-19 boosters.

The FDA has amended the emergency use authorizations (EUAs) of both the updated Moderna and the Pfizer/BioNTech COVID-19 booster vaccines that include the omicron variants. The updated boosters are bivalent vaccines and contain two messenger RNA (mRNA) components, one of the original strain of the SARS-CoV-2 and the BA.1, BA.4 and BA.5 omicron variant.

The Moderna booster is a single dose in people 18 years of age and older. The Pfizer-BioNTech booster is a single dose in people 12 years of age and older. People are eligible for the updated boosters if it’s been at least two months since they have had most recent booster or the primary vaccination series.

Pfizer had indicated last week when it submitted its EUA that the updated booster would be available to ship immediately. Moderna indicated that its updated booster would be ready to ship in the next few days, the company said in a press release.

FDA approves Xenpozyme for ASMD, a rare genetic disorder.

The FDA has approved Sanofi’s Xenpozyme (olipudase alfa-rpcp) to treat both adults and children with acid sphingomyelinase deficiency (ASMD). Xenpozyme is an intravenous infusion used for the treatment of non-central nervous system (non-CNS) manifestations of the disease. Xenpozyme is expected to be available in the United States in the coming weeks. The wholesale acquisition cost of Xenpozyme of $7,142 per vial, according to Sanofi.

ASMD, historically known as Niemann-Pick disease types A, A/B, and B, is a rare genetic disorder where a deficiency of the enzyme acid sphingomyelinase results in sphingomyelin accumulating in various tissues in the body. The most severely affected patients have profound neurologic symptoms and rarely survive beyond two to three years of age. Other patients may survive into adulthood but die prematurely from respiratory failure. It has been estimated that there are fewer than 120 patients diagnosed with ASMD in the United States. About two-thirds of patients with ASMD in the United States are children.

Xenpozyme is designed to replace deficient or defective acid sphingomyelinase (ASM). Xenpozyme is not expected to cross the blood-brain barrier or modulate CNS manifestations of ASMD. The therapy has not been studied in patients with ASMD type A.

FDA approves first targeted treatment for myeloid/lymphoid neoplasms.

The FDA has approved Incyte’s Pemazyre (pemigatinib) to treat adults with relapsed or refractory myeloid/lymphoid neoplasms (MLNs). Pemazyre is a selective fibroblast growth factor receptor (FGFR) inhibitor that specifically targets FGFR1 rearrangement, an extremely rare and aggressive blood cancer.

Pemazyre is also approved under an accelerated approval based on overall response rate and duration of response to treat adults with bile duct cancer that has spread or cannot be removed by surgery. The wholesale acquisition cost is $17,510 for a 14-count bottle, according to Incyte.

FDA expands Imbruvica for young children with GVHD.

The FDA has approved Imbruvica (ibrutinib) to treat children one year and older with chronic graft versus host disease (cGVHD) after failure of one or more lines of systemic therapy. This is the first pediatric indication for Imbruvica and the 12th approval for the therapy.

The recommended dose for patients 12 years and older is 420 mg taken orally once daily. In patients one year old to less than 12 years of age, the recommended dose is 240 mg/m2 taken orally once daily (up to a dose of 420 mg). This FDA approval also includes an oral suspension formulation of Imbruvica.

Imbruvica is jointly developed and commercialized by Janssen Biotech and Pharmacyclics, an AbbVie company.

FDA approves novel treatment for rare type of psoriasis flare.

The FDA has approved Boehringer Ingelheim’s Spevigo (spesolimab-sbzo) injection, the first treatment option for adults with generalized pustular psoriasis (GPP) flares. Spevigo is a novel antibody that blocks the activation of the interleukin-36 receptor (IL-36R), a key part of a signaling pathway within the immune system shown to be involved in the cause of generalized pustular psoriasis.

Generalized pustular psoriasis is different from plaque psoriasis. It is a rare and potentially life-threatening neutrophilic skin disease, which is characterized by flares. In the United States, it is estimated that one out of every 10,000 people has generalized pustular psoriasis.

In the 12-week pivotal Effisayil 1 clinical trial, 53 patients experiencing a generalized pustular psoriasis flare were treated with Spevigo or placebo. After one week, 54% of patients treated with Spevigo showed no visible pustules compared with 6% in the placebo in the placebo arm. The most common adverse reactions in patients who received Spevigo were asthenia and fatigue, nausea and vomiting, headache, pruritus and prurigo, infusion site hematoma and bruising, and urinary tract infection.

FDA grants priority review to weekly hemophilia A therapy.

The FDA has accepted for priority review the biologics license application (BLA) for efanesoctocog alfa (BIVV001) for the treatment of hemophilia A, a rare bleeding disorder. The target action date for the FDA decision is Feb. 28, 2023.

Sanofi and Sobi collaborated on the development and commercialization of efanesoctocog alfa. Efanesoctocog alfa is a novel recombinant factor VIII therapy that is designed to extend protection from bleeds with once-weekly prophylactic dosing for people with hemophilia A.

The BLA was based on the XTEND-1 phase 3 study, which found that efanesoctocog alfa demonstrated a clinically meaningful prevention of bleeds in people with severe hemophilia A over a 52-week period. Efanesoctocog alfa was able to deliver high sustained factor activity levels in the normal to near-normal range for the majority of the week with once weekly prophylaxis dosing.