Product Approvals and Launches

The approval makes atidarsagene autotemcel (arsa-cel [Lenmeldy]; Orchard Therapeutics) the first approved treatment for metachromatic leukodystrophy, a disease caused by a mutation in the ARSA gene and marked by progressive declines in both the central and peripheral nervous systems.

In a recent issue of JAMA, a team led by Benjamin N. Rome, MD, MPH, examined how the FDA’s accelerated approval process has moved 5 genetically targeted treatments for Duchenne muscular dystrophy (DMD) through its pipeline despite limited evidence on their efficacy.

The FDA has approved tislelizumab-jsgr (Tevimbra) for single-agent use in adult patients with unresectable or metastatic esophageal squamous cell carcinoma following prior systemic chemotherapy that did not include a PD-1/PD-L1 inhibitor.

The approval fills a void for patients with CLL or SLL whose disease progresses after treatment with a BTK inhibitor and a BCL-2 inhibitor; until now, there has been no standard of care.

Since 2016, 5 targeted treatments have received accelerated approval from the FDA for use in Duchenne muscular dystrophy (DMD), and the total spend for just 3 of them is $3.1 billion.

The first medication for the treatment of nonalcoholic steatohepatitis (NASH) and liver fibrosis received accelerated approval. Previously, the only treatment available to patients was implementing lifestyle changes aimed at weight reduction.

March 1 saw the FDA grant full approval to amivantamab plus chemotherapy for first-line use in patients who have non–small cell lung cancer (NSCLC) with EGFR exon 20 insertion mutations, converting the May 2021 accelerated approval for this treatment in the second line.

Teclistamab (Tecvayli, Johnson & Johnson), a first-in-class bispecific T-cell–engaging antibody, is approved to treat relapsed/refractory multiple myeloma in the US.

The FDA added another indication for semaglutide (Wegovy), expanding its use to reduce the risk of cardiovascular death, heart attack, or stroke in adults who have cardiovascular disease (CVD) and overweight or obesity.

The FDA granted accelerated approval to combination zanubrutinib and obinutuzumab for the treatment of relapsed or refractory (R/R) follicular lymphoma following 2 or more lines of therapy.

The FDA approval of nivolumab plus cisplatin and gemcitabine marks the first concurrent immunotherapy-chemotherapy combination for the population.

Starting summer 2024, individuals across the US will be able to purchase a continuous glucose monitor (CGM) without needing a prescription.

Wyost and Jubbonti were approved as interchangeable biosimilars for Xgeva and Prolia; however, there is no launch date yet pending litigation with the maker of the reference products.

The approval converts the May 2021 accelerated FDA approval for second-line treatment of non–small cell lung cancer (NSCLC) with EGFR exon 20 insertion mutations to a full approval in the first line following results from the phase 3 PAPILLON study.

The FDA recently approved the supplemental biologics license application for teclistamab (Tecvayli; Johnson & Johnson) to treat relapsed/refractory multiple myeloma on a biweekly dosing schedule.

Teclistamab (Tecvayli) was first approved for use in patients who have relapsed/refractory multiple myeloma (RRMM) in October 2022, making it a first-in-class bispecific antibody.

The bispecific antibody is used to treat patients with relapsed or refractory multiple myeloma (R/R MM) who have achieved and maintained a complete response for at least 6 months; this approval allows a dosing frequency of 1.5 mg/kg every 2 weeks.

Besides the new approval, AstraZeneca released preliminary results for LAURA evaluating osimertinib after chemoradiotherapy.

Lifileucel was granted approval for the treatment of adults with unresectable or metastatic melanoma that has been previously treated with other therapies, marking the first approval of a tumor-derived cellular therapy in a solid tumor cancer.

A tumor-agnostic approval would be a significant development in the advance of antibody-drug conjugates (ADCs), which are designed to deliver a potent, cancer-fighting payload into a tumor while sparing nearby tissues.

The expanded indication makes dupilumab the only approved treatment for eosinophilic esophagitis (EoE) in this age group.

CMS has announced new federal rules that require health insurers to streamline requests to cover treatments; nearly 50,000 veterans used the emergency suicide prevention program launched by the Department of Veteran Affairs in 2023; the FDA recently authorized the first artificial intelligence (AI)-powered medical device to help doctors detect the most common forms of skin cancer.

The FDA and the European Medicines Agency have accepted marketing authorization applications and decisions are pending.

Bio-Thera Solutions’ Avzivi (bevacizumab-tnjn) has become the fifth biosimilar referencing Avastin (bevacizumab) to be approved in the United States.

The human monoclonal antibody known as tralokinumab-ldrm was tested and approved as a safe, effective treatment for pediatric patients with atopic dermatitis aged 12 to 17 years old.

In the phase 3 EV-302/KEYNOTE-A39 clinical trial, the antibody-drug conjugate enfortumab vedotin plus PD-1 inhibitor pembrolizumab nearly doubled both median overall survival and progression-free survival compared with chemotherapy.

The approval of belzutifan, an oral hypoxia-inducible factor-2 alpha inhibitor, provides a new option for patients with advanced renal cell carcinoma (RCC) following treatment with a programmed death receptor-1 (PD-1) or programmed death-ligand 1 (PD-L1) inhibitor and a vascular endothelial growth factor tyrosine kinase inhibitor (VEGF-TKI).

Colin Howden, MD, discusses the mechanisms at work in vonoprazan as well as patient monitoring and potential future uses following a recent FDA approval for the treatment of erosive esophagitis.

The decision was supported by efficacy data from 36 patients from the ongoing phase 1/2 HGB-206 trial (NCT02140554) and 2 patients in the phase 3 HGB-210 trial (NCT04293185). It was the second approval from FDA for a gene therapy for sickle cell disease.

The FDA has approved a gene-editing therapy for sickle cell disease (SCD) for the first time.