Articles related to DEVICE/DRUG APPROVALS

According to Aimee Tharaldson, PharmD, a senior clinical pharmacist who spoke at the AMCP Nexus 2022 meeting, specialty medications continue to lead FDA approvals, with nearly half being developed for rare diseases. In 2023, many candidates will seek an orphan drug indication, which is a special FDA designation that provides tax credits for qualified clinical trials, exemption from user fees, and up to seven years of market exclusivity after approval. An estimated 7,000 orphan diseases exist.

Approved for AD in adults a year ago, abrocitinib is now available for individuals over 12 years old with moderate to severe atopic dermatitis that is not adequately controlled with other systemic therapy.

The SGLT2 inhibitor bexagliflozin is now available under the brand name Brenzavvy. Approval was based on monotherapy trials versus glimepiride, where the new drug was non-inferior, and in combination with metformin.

Emzahh (norethindrone tablets), the progestin-only generic equivalent of the discontinued Ortho Micronor, was approved via an abbreviated NDA.

Based on prior studies in patients with melanoma and lung cancer as well as overall response and duration of response rates in open-label, multiple cohort trials involving 131 adults and 36 children, the combination was approved for the new indication (treatment of unresectable or metastatic solid tumors with a BRAF V600E mutation in patients ≥6 years old who have progressed following prior treatment and have no satisfactory alternative treatment options). Adults with BRAF V600E mutation-positive solid tumors, such as low-grade glioma (LGG), anaplastic thyroid cancer, biliary tract cancer, small intestine cancer, and high-grade glioma (HGG), were enrolled in the new trials, along with 36 children with BRAF V600 refractory or recurrent LGG or HGG. The overall response rate (ORR) served as the trial’s main measure of success, and out of the 131 adults who participated, 54 (41%) had an objective reaction. Among the most prevalent tumor types in the clinical trials, the ORR for biliary tract cancer was 46%, HGG was 33%, and LGG was 50%. Children’s ORR was 25%.

The approval is based on the findings of the DESTINY-Breast04 Phase III trial and follows the Committee for Medicinal Products for Human Use’s favorable recommendation. In the trial, Enhertu (trastuzumab deruxtecan) significantly decreased the risk of disease progression or death in patients with HER2-low metastatic breast cancer with hormone receptor (HR)-positive or HR-negative disease by 50% compared to the physician’s choice of chemotherapy. According to a blinded independent central evaluation, Enhertu had a median progression-free survival (PFS) of 9.9 months compared to chemotherapy patients’ PFS of 5.1 months (BICR). When compared to chemotherapy, Enhertu showed a 36% reduction in the risk of death, with a median overall survival (OS) of 23.4 months as opposed to 16.8 months.