Product Approvals and Launches

The first-in-class oral factor D inhibitor danicopan (Voydeya) has been approved for the management of extravascular hemolysis in patients with paroxysmal nocturnal hemoglobinuria (PNH).

Ravulizumab-cwvz (Ultomiris) received a label expansion by the FDA for the treatment of adult neuromyelitis optica spectrum disorder in patients with anti–aquaporin-4 antibodies after trials results showed it could prevent relapses.

The FDA approved vadadustat (Vafseo), an oral medication, to treat anemia in adult patients with chronic kidney disease (CKD) on dialysis for at least 3 months. This fills a need for a new treatment option as anemia is common in these patients and can significantly impact their quality of life.

Adults with pulmonary arterial hypertension (PAH) have a new treatment available with the FDA approval of sotatercept (Winrevair; Merck), an activin signaling inhibitor biologic.

The approval, which marks the first for a once-daily, single-tablet combination therapy for pulmonary arterial hypertension, is based on findings from the phase 3 A DUE study.

This expansion applies to the estimated 70 million patients who need either primary or secondary prevention, regardless of statin use.

Mirvetuximab soravtansine-gynx (Elahere) received full FDA approval for the treatment of folate receptor alpha–positive (FRα+), platinum-resistant ovarian cancer (PROC) based on findings from the confirmatory phase 3 MIRASOL trial.

The approval follows fast track, orphan drug, and rare pediatric designations and an original Prescription Drug User Fee Act date of December 21, which the FDA pushed back to March 21 to have more time to review Italfarmaco’s application.

Once-daily aprocitentan (Tryvio) is the first and only FDA-approved endothelin receptor antagonist for treating high blood pressure that remains uncontrolled despite other antihypertensive treatments.

The Duchenne muscular dystrophy (DMD) treatment vamorolone received FDA approval in October 2023 and is a less toxic alternative to conventional corticosteroids.

The FDA has granted accelerated approval to ponatinib (Iclusig) plus chemotherapy for the first-line treatment of Philadelphia chromosome–positive acute lymphoblastic leukemia (Ph+ ALL).

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.