Product Approvals and Launches

Daprodustat (Jesduvroq, GSK) is the first oral hypoxia-inducible factor prolyl hydroxylase inhibitor to gain approval in the United States.

The approval is based on results from the phase 3 TROPiCS-02 trial demonstrating overall and progression survival benefits with sacituzumab govitecan compared with physician’s choice of single-agent chemotherapy.

The FDA has approved pirtobrutinib (Jaypirca) for the treatment of adult patients with relapsed or refractory mantle cell lymphoma following at least 2 lines of systemic therapy, including a previous Bruton tyrosine kinase (BTK) inhibitor.

The FDA has approved elacestrant (Orserdu) for the treatment of postmenopausal women or adult men with estrogen receptor–positive, HER2-negative, ESR1-mutated advanced or metastatic breast cancer with disease progression following at least 1 line of endocrine therapy.

The broad approval, while expected, will likely signal a milestone in the treatment of chronic lymphocytic leukemia (CLL) and small lymphocytic lymphoma (SLL).

The combination of tucatinib and trastuzumab received accelerated approval from the FDA to treat adults with RAS wild-type, HER2-positive unresectable or metastatic colorectal cancer (mCRC) that advances after treatment with specific types of chemotherapy.

Ublituximab-xiiy, developed by TG Therapeutics, will be sold under the name Briumvi.

The FDA’s approval of lecanemab for Alzheimer disease is very important, but there will remain significant challenges around pricing and access that will need to be addressed to deliver on the promise the therapy actually represents, said Alvaro Pascual-Leone, MD, PhD, of Harvard Medical School.

The lecanemab decision follows the controversial 2021 approval of aducanumab, which led to tight coverage restrictions for monoclonal antibodies directed against amyloid for the treatment of Alzheimer disease.

Jessica Allegretti, MD, MPH, medical director of the Crohn's and Colitis Center, Brigham and Women's Hospital, explained the mechanism of action for Rebyota, the first fecal transplant therapy approved by the FDA for the prevention of recurrent Clostridioides difficile infection (CDI) in individuals 18 years and older, following antibiotic treatment for recurrent CDI.

Ferring’s Rebyota, a novel first-in-class microbiota-based live biotherapeutic, has been approved by the FDA for the prevention of recurrence of Clostridioides difficile infection (CDI) in individuals 18 years and older, following antibiotic treatment for recurrent CDI.

The biologic is the first drug that aims to delay the diagnosis of type 1 diabetes.

In addition to the drug approval, the FDA approved a companion diagnostic to be used to identify patients who are eligible to receive the treatment.

The FDA approved cemiplimab (Libtayo) in combination with platinum-based chemotherapy for the first-line treatment of advanced non–small cell lung cancer (NSCLC) with no EGFR, ALK or ROS1 aberrations.

The FDA’s approval of teclistamab for relapsed/refractory (R/R) multiple myeloma makes it the first bispecific T-cell engager antibody to enter the treatment landscape.

A dual immunotherapy option of tremelimumab, sold as Imjudo, in combination with durvalumab, sold as Imfinzi, was approved by the FDA Monday for the treatment of adult patients with unresectable hepatocellular carcinoma, the most common type of liver cancer.

New and emerging medications in the specialty drug pipeline were discussed during a keynote address at AMCP Nexus 2022 by Aimee Tharaldson, PhD, senior clinical pharmacist of emerging therapeutics at Express Scripts, with biosimilars, orphan drugs, and cell and gene therapies emerging as key trends to watch.

The targeted therapy, to be sold by Taiho Oncology as Lytgobi, had previously received breakthrough status in 2021 and was accepted for priority review in March.

The gene therapy is the first treatment for active cerebral adrenoleukodystrophy, a fatal neurodegenerative disease that affects boys.

An estimated 1 in 4 patients treated with durvalumab and chemotherapy (gemcitabine plus cisplatin) was alive at 2 years compared with 1 in 10 treated with chemotherapy alone, with these results contributing to the FDA's approval of the first immunotherapy to treat these cancers.

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.