Product Approvals and Launches

FDA has given accelerated approval for lisocabtagene maraleucel (liso-cel; Breyanzi) for patients with relapsed or refractory (R/R) follicular lymphoma.

In part 2 of our interview with Binod Dhakal, MD, he addresses how CARTITUDE-4 study findings help to advance the clinical understanding of ciltacabtagene autoleucel (cilta-cel) and the importance of vigilance and education on the treatment’s adverse effects.

The FDA granted full approval for tisotumab vedotin-tftv to treat recurrent or metastatic cervical cancer that progressed after chemotherapy, offering patients improved overall survival and a manageable safety profile.

The FDA has approved fidanacogene elaparvovec-dzkt (Beqvez; Pfizer), a gene therapy administered in a one-time dose, for certain patients with moderate to severe hemophilia B.

The FDA recently approved an antibiotic for the treatment of urinary tract infections (UTIs) in women; a Senate committee recently launched an investigation into the prices of Novo Nordisk’s diabetes and weight loss drugs; US births fell last year, resuming a national slide after a previous increase during the pandemic.

Binod Dhakal, MD, associate professor, Medical College of Wisconsin, is lead investigator of the CARTITUDE-4 study, from which data were used by the FDA to approve ciltacabtagene autoleucel (cilta-cel) for patients with relapsed/refractory multiple myeloma (RRMM) who have received at least 1 prior line of therapy.

The principal investigator of the study leading to approval said this new immunotherapy could be a "game changer" in bladder cancer.

The approval of benralizumab (Fasenra) is supported by evidence from the phase 3 TATE study.

Idecabtagene vicleucel (ide-cel) was able to triple progression-free survival and reduce the risk of disease progression or death in a phase 3 trial.

Vanda Pharmaceuticals announces the FDA approval of iloperidone (Fanapt) for the acute treatment of bipolar 1 disorder.

Eko Health's artificial intelligence (AI) tool can detect low ejection fraction in seconds via an Eko stethoscope, bringing earlier heart failure detection to routine checkups.

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.