Product Approvals and Launches

The FDA Friday approved pemigatinib (Pemazyre), a selective fibroblast growth factor (FGFR) inhibitor, to treat adults who have relapsed or refractory (R/R) myeloid/lymphoid neoplasms (MLNs) with FGFR1 rearrangement, a very rare and aggressive cancer.

The FDA Wednesday approved betibeglogene autotemcel (Zynteglo), the first cell-based gene therapy for the treatment of adult and pediatric patients with beta-thalassemia who require regular red blood cell transfusions.

Darolutamide, sold as Nubeqa, is an androgen receptor inhibitor, which works to limit prostate cell growth by preventing the binding of hormones, or androgens, to proteins known as androgen receptors.

The approval came exactly 2 months after results from the landmark DESTINY-Breast04 trial showed that the antibody drug conjugate reduced the risk of disease progression or death by 50% compared with chemotherapy for HER2-low patients with both hormone receptor (HR)–positive and HR-negative disease.

Ruxolitinib (Opzelura) cream is the first and only FDA-approved treatment for repigmentation in patients with vitiligo.

Pegloticase (Krystexxa) injection coadministered with methotrexate received FDA approval for the treatment of gout.

The combination is the first and only therapy to be approved with a tumor-agnostic indication in both children and adults with solid tumors that have a BRAF V600E mutation, Novartis said late Wednesday.

The FDA approval for Fylnetra represents the fifth approval for a biosimilar referencing Neulasta (pegfilgrastim) in the United States.

Tisagenlecleucel (tisa-cel) was approved to treat adults with relapsed or refractory (R/R) follicular lymphoma. This is the third indication for the therapy since it became the first FDA-approved chimeric antigen receptor T-cell therapy in 2017.

The approvals were announced by Bristol Myers Squibb (BMS), maker of the 2 immunotherapy treatments approved for 1 combination: nivolumab (Opdivo), the first approved PD-1 immune checkpoint inhibitor, and ipilimumab (Yervoy), which activates the immune system by targeting CTLA-4.

Eosinophilic esophagitis is a chronic inflammatory disorder in which eosinophils, a type of white blood cell, proliferate in the esophagus, causing difficulties with eating and swallowing.

The treatment was found to improve glycemic control and is indicated to be used in conjunction with diet and exercise.

This approval of trastuzumab deruxtecan (Enhertu) follows the March release of data from the DESTINY-Breast03 trial on the anti-HER2 monoclonal antibody, also a HER2-directed antibody-drug conjugate, which indicated superior outcomes vs trastuzumab emtansine.

The drug, oteseconazole, is an azole antifungal indicated to reduce the incidence of recurrent vulvovaginal candidiasis in females who are infertile or postmenopausal.

A new drug has been approved for obstructive hypertrophic cardiomyopathy (HCM); Oklahoma enacts a strict abortion ban; whistleblower claims alarms had been raised about Abbott baby formula contamination.

Alymsys from Amneal Pharmaceuticals and mAbxience is the third biosimilar referencing Avastin approved in the United States and the second of 3 biosimilar approvals Amneal expects in 2022.

The expanded use of axi-cel, sold as Yescarta, while not unexpected, nonetheless represents uncharted territory in cancer care.

The FDA approved a drug to treat a rare form of childhood epilepsy; uninsured Americans will no longer have access to free COVID-19 tests; a conservation group is suing the Environmental Protection Agency (EPA) over failure to protect rivers from pollution.

Full approval by the FDA follows tentative approval for the complex drug delivery advice for asthma and chronic obstructive pulmonary disease (COPD) granted by the FDA last week amid a patent dispute.

The FDA based its approval on OlympiA trial data, which show the PARP inhibitor has the ability to reduce risks of invasive breast cancer recurrence, second cancers, and death, and to improve overall survival.

Steven Yeh, MD, professor of ophthalmology at the Truhlsen Eye Institute at the University of Nebraska Medical Center, explains the delivery mechanism and benefits of Xipere for macular edema.

Releuko, a filgrastim biosimilar developed by Kashiv Biosciences and Amneal Pharmaceuticals, becomes the third filgrastim biosimilar to be approved by the FDA.

Steven Feldman, MD, PhD, professor of dermatology at Wake Forest School of Medicine, discusses what makes clascoterone (Winlevi) different from past acne treatments.

FDA approval is based on CARTITUDE-1, a phase 1b/2 trial in which investigators reported that cilta-cel produced an objective response rate (ORR) of 98% and a stringent complete response rate of 78%.

Jardiance (empagliflozin) received approval from the FDA for a new indication to treat heart failure in a broader range of patients, including those with preserved ejection fraction.

The treatment was approved in October 2021 and is the first FDA-approved therapy to treat macular edema associated with uveitis via injection to the suprachoroidal space, which provides a more targeted delivery mechanism.

Foundation Medicine’s circulating tumor DNA (ctDNA) detection and monitoring assay, FoundationOne Tracker, was granted a Breakthrough Device Designation from the FDA, streamlining the approval and review processes to give patients and providers earlier access to the device.

The drug will be marketed by Sanofi under the name Enjaymo; it is the first treatment approved for cold agglutinin disease.

The JAK1 inhibitor abrocitinib (Cibinqo) was recently approved by the FDA for the treatment of adults with refractory, moderate to severe atopic dermatitis.

Americans who are boosted are 95 times less likely than unvaccinated people to die of COVID-19, the FDA approved the first generic drug for Restasis to treat dry eye syndrome, and CMS is putting a greater focus on health equity for Medicare Advantage and Part D plans.