Politics and Policy

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.

Commercial shipments are already under way of Cosette’s gel formulation of tazarotene to treat patients with acne and facial wrinkles.

Veru is seeking an emergency use authorization for sabizabulin, a first-in-class treatment for hospitalized COVID-19 patients at high risk for acute respiratory distress syndrome. The advisory committee meeting is now Nov. 9, 2022.

There are currently no FDA-approved therapies that specifically target HER2 in colorectal cancer. The agency has set a target action date of Jan. 19, 2023.

Skysona, with a wholesale acquisition cost of $3 million, is the first FDA approved therapy to slow progression of neurologic dysfunction in boys with this neurodegenerative disease.

Aponvie is the same formulation as Cinvanti, which is an injectable emulsion for prevention of chemotherapy-induced nausea and vomiting.

The applications are based on progression-free survival data even though the FDA has recommended that the company wait for more mature overall survival data.

Terlivaz is the first approved therapy to treat hepatorenal syndrome, a rare form of kidney disfunction related to advanced liver disease.

Aripiprazole, currently available as a once-monthly injection under the brand name Abilify Maintena, is being reviewed as a two-month therapy. The FDA target date for completion of the review is April 27, 2023.

The FDA has granted priority review of velmanase alfa, an enzyme replacement, to treat patients with alpha-mannosidosis, a rare genetic disease. The FDA has assigned an action date in the first half of 2023.