Regulatory Updates

Novavax’ protein-based vaccine is engineered from the genetic sequence of the first strain of SARS-CoV-2, the virus that causes COVID-19 disease.

If approved, tofersen will be the first treatment that targets a genetic cause of ALS. The updated PDUFA date is April 25, 2023.

If approved, valoctocogene roxaparvovec would be the first gene therapy in the U.S. for the treatment of severe hemophilia A. A PDUFA action date has been set for March 31, 2023.

The bivalent booster vaccines add the omicron variant BA.4 and BA.5 to the original SARS-CoV-2 along with a component of omicron BA.1.

The FDA has assigned a target action date of Feb. 11, 2023.

The submission is expected to be complete by the end of the year, and if approved, could be available in early 2024.

The FDA is asking for additional analysis related to the infusion device used with SPN-830, which contains apomorphine to treat patients with Parkinson’s disease.

Furoscix can be administered at home with the use of the On-Body Infusor, which delivers furosemide over five hours. It will be launched in the first quarter of 2023.

Regulators used a re-analysis of data from an observational case-control study of Tdap vaccine effectiveness to show that Boostrix given in the third-trimester prevented pertussis among infants.

This approval for Oxlumo adds lowering plasma oxalate levels for patients with primary hyperoxaluria type 1, a rare and life-threatening metabolic disease.

The FDA had indicated that it would not issue an emergency use authorization without additional data for peginterferon lambda to treat patients with mild-to-moderate COVID-19.

The FDA is expected to make a decision on approval in this patient population in the first half of 2023.

Medicare and Medicaid spent more than $18 billion on drugs without verified clinical benefit.

Trogarzo was approved with a new method of administration designed to make maintenance dosing easier for patients and healthcare providers.

If approved, valoctocogene roxaparvovec would be the first commercially-available gene therapy in the United States for the treatment of severe hemophilia A.

Taiho’s Lytgobi is approved to treat advanced or metastatic advanced bile duct cancer in patients with FGFR2 gene fusions.

Now with the brand name Relyvrio, the new therapy should be available within four to six weeks. It will have a wholesale acquisition cost of $158,000 per year.

Dupixent is the first treatment approved to treat prurigo nodularis, a chronic skin disorder characterized by the presence of hard, itchy nodules.

Vegzelma is Celltrion’s third oncology biosimilar to be approved by the FDA.

The FDA has assigned a Prescription Drug User Fee Act (PDUFA) goal date of March 29, 2023, for a decision on leniolisib as a treatment for APDS, a rare primary immunodeficiency.

Omlonti is approved to reduce elevated intraocular pressure in patients with primary open-angle glaucoma or ocular hypertension

Bebtelovimab is available under an emergency use authorization, and the HHS is making doses available to those who are uninsured and underinsured.

Committee members felt the data on overall survival for patients treated with Copiktra was difficult to interpret, and the therapy was associated with a higher risk of serious side effects and deaths compared with Kesimpta.

The companies have also initiated a phase 1/2/3 study to evaluate different doses and dosing regimens of the BA.4/BA.5-adapted bivalent COVID-19 vaccine in children 6 months through 11 years of age.

While some patients with relapsed or refractory multiple myeloma saw a benefit in a confirmatory trial, the main issue concerning the committee members was a high rate of death in the study.

RBX2660 — now with the brand name Rebyota — is a potential first-in-class microbiota-based live biotherapeutic that aims to reduce recurrent C. difficile infection.

Several concerns factored into the decision, including a low overall response rate and high rate of adverse events, a dose that was not optimized and a confirmatory trial that will not likely see results until 2026.

This is the first RET Inhibitor for adults with advanced or metastatic solid tumors with a RET gene fusion, regardless of tumor type.

Elucirem requires half the gadolinium dose of existing gadolinium-based contrast agents, addressing practitioners’ concerns about gadolinium exposure during MRIs. It will be available in bottle and pre-filled syringe form.

World Health Organization said there is a lack of evidence of clinical effectiveness of the monoclonal antibodies Xevudy and Regen-Cov against the COVID-19 variants that are currently circulating.