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FDA Updates: First Lupus Drug Approved in a Decade and Another Accelerated Approval Drug Loses An Indication


AstraZeneca gets FDA nod for Saphnelo, BMS pulls indication for Istodax, Merck to seek another use for Keytruda, Tecentriq is granted priority review for NSCLC and COVID-19 cocktail gets expanded EUA.

FDA approves AstraZeneca lupus drug

The FDA has approved Saphnelo (anifrolumab-fnia) for the treatment of adult patients with moderate-to-severe systemic lupus erythematosus (SLE) who are receiving standard therapy.

The agency's OK marks the first regulatory approval for a type I interferon (type I IFN) receptor antagonist and the first new treatment approved for SLE in more than decade.

BMS pulls lymphoma indication for Istodax

Bristol Myers Squibb has withdrawn the indication for Istodax (romidepsin) as a monotherapy for the treatment of peripheral T-cell lymphoma (PTCL) in adult patients who have received at least one prior therapy. In 2011, Celgene, now a subsidiary of BMS, received accelerated approval for this indication. The approval was based on results from two clinical studies that assessed the effect of Istodax on the surrogate end point of overall response rate.

Bristol Myers Squibb conducted a confirmatory phase 3 study evaluating Istodax plus a combination regimen of cyclophosphamide, doxorubicin, vincristine, and prednisone (CHOP) versus just CHOP as a first-line treatment of PTCL patients. Results from the trial showed that Istodax did not meet the primary end point of progression-free survival.

Merck submits application for another Keytruda indication

The FDA has accepted Merck’s supplemental Biologics License Application and granted Priority Review for Keytruda (pembrolizumab) as an adjuvant treatment for adult and pediatric (12 years and older) patients with stage IIB or IIC melanoma. Keytruda is already approved to treat patients with resected stage III melanoma. The PDUFA date for this indication is December 4, 2021.

This application is based on the phase KEYNOTE-716 trial in which Keytruda met its primary end point of recurrence-free survival (RFS) in stage II melanoma. An interim analysis showed that treatment with Keytruda as a single agent was associaed with a statistically significant and clinically meaningful improvement in RFS compared with placebo as adjuvant therapy for these patients. No new safety problems were observed. Merck officials said results will be presented at an upcoming medical meeting.

FDA grants Tecentriq priority review for NSCLC

The FDA has granted priority review for Roche’s Tecentriq (atezolizumab) as an adjuvant treatment following surgery and platinum-based chemotherapy for people with nonsmall cell lung cancer (NSCLC) whose tumors express PD-L1. The FDA is reviewing Roche’s supplemental Biologics License Application application and is expected to make a decision on approval by December 1, 2021.

Tecentriq is approved in the United States, European Union, and countries around the world, either alone or in combination with targeted therapies and/or chemotherapies in various forms of NSCLC, small cell lung cancer, certain types of metastatic urothelial cancer, in PD-L1-positive metastatic triple-negative breast cancer and for hepatocellular carcinoma.

New therapy for Pompe disease is approved

The FDA has approved Genzyme’s Nexviazyme (avalglucosidase alfa-ngpt) for intravenous infusion to treat patients 1 year of age and older with late-onset Pompe disease. Patients with Pompe disease have an enzyme deficiency that leads to the accumulation of glycogen in skeletal and heart muscles tissue that causes muscle weakness and premature death from respiratory or heart failure.

Nexviazyme, an enzyme replacement therapy, is an intravenous medication that helps reduce glycogen accumulation.

Genzyme is a subsidiary of Sanofi.

Exelixis submits Cabometyx for thyroid cancer

The FDA has accepted the supplemental New Drug Application for Exelixis’ Cabometyx (cabozantinib) as a treatment for patients 12 years and older with differentiated thyroid cancer who have progressed following prior therapy and are radioactive iodine-refractory.

The FDA granted Priority Review designation and assigned a PDUFA target action date of December 4, 2021.

The application is based on the results of COSMIC-311, a phase 3 pivotal trial evaluating Cabometyx. The results showed that the therapy met one of the trial’s primary end points, demonstrating a significant improvement in progression-free survival compared with placebo. At a planned interim analysis, the therapy demonstrated a significant reduction in the risk of disease progression or death of 78% versus placebo.

FDA grants breakthrough designation for avexitide

Eiger BioPharmaceuticals has received Breakthrough Therapy Designation for avexitide for the treatment of congenital hyperinsulinism. Eiger’s application was supported by data from three completed phase 2 studies in 39 infants, children, and adolescents with congenital hyperinsulinism.

Avexitide is a targeted, first-in-class, GLP-1 antagonist in development for the treatment of metabolic disorders, including congential hyperinsulinism, a rare, life-threatening, pediatric disorder of persistent hypoglycemia that results in irreversible brain damage in up to 50% of children.

Avexitide has also been granted Orphan Drug Designation by the FDA for the treatment of hyperinsulinemic hypoglycemia (which includes congenital hyperinsulinism), Orphan Drug Designation by the European Medicines Agency for the treatment of congenital hyperinsulinism and Rare Pediatric Disease Designation by the FDA.

Regeneron’s COVID-19 therapy gets expanded EUA

The FDA has updated Regeneron Pharmaceuticals’ Emergency Use Authorization (EUA) for the investigational COVID-19 antibody cocktail, REGEN-COV, which a combination of casirivimab and imdevimab.

REGEN-COV can now be used for post-exposure prophylaxis in people at high risk for progression to severe COVID-19, who are not fully vaccinated or are not expected to mount an adequate response to vaccination and have been exposed to someone with COVID-19 or are at high risk of exposure to an infected individual because of infection occurring in the same institutional setting.

This new indication in people aged 12 and older is in addition to the previously granted authorization to treat nonhospitalized patients.

Spectrum gets Complete Response Letter for Rolontis

Spectrum Pharmaceuticals has received a Complete Response Letter (CRL) from the FDA about its Biologics License Application (BLA) for Rolontis (eflapegrastim). The CRL cited deficiencies related to manufacturing and indicated that a reinspection will be necessary.

Rolontis is a long-acting granulocyte colony-stimulating factor (G-CSF) seeking an indication for the treatment of neutropenia in patients receiving myelosuppressive anti-cancer drugs, which can lead to a risk of infections. The company’s application is supported by data from two identically designed phase 3 clinical trials, which evaluated the safety and efficacy of Rolontis in 643 early-stage breast cancer patients. In both studies, the therapy demonstrated noninferiority in duration of severe neutropenia and a similar safety profile to pegfilgrastim.

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