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FDA Updates for Week of April 1, 2024: Approvals for Blood Disorder and Bacterial Infections


The FDA has approved several new therapies this week, including Voydeya as an add-on therapy for blood disorder PNH; Zevtera for serious bacterial infections; Fanapt for bipolar 1; and the CAR-T therapy Abecma for earlier treatment in multiple myeloma. The agency has also accepted a supplemental BLA for Bimzelx in hidradenitis suppurativa and set a review date for datopotamab deruxtecan in breast cancer.

FDA Approves Voydeya as Add-on Therapy in Rare Blood Disorder

The FDA has approved Alexion/AstraZeneca’s Voydeya (danicopan) as add-on therapy to Ultomiris (ravulizumab) or Soliris (eculizumab) to treat adult patients with paroxysmal nocturnal hemoglobinuria (PNH). It is indicated for those patients with extravascular hemolysis (EVH) where red blood cells are destroyed outside of the blood vessel despite treatment with Soliris or Ultomiris.

PNH is a rare, chronic and progressive blood disorder. It is characterized by red blood cell destruction within blood vessels and white blood cell and platelet activation, which can result in thrombosis.

Voydeya is a first-in-class, oral, Factor D inhibitor developed as an add-on to standard-of-care Solis or Ultomiris to address the needs of about 10% to 20% of patients with PNH who experience clinically significant extravascular hemolysis while treated with a C5 inhibitor. The companies have not indicated when Voydeya will be available or what the list price is.

The approval of Voydeya was based on positive results from the pivotal ALPHA phase 3 trial, which showed a clinically meaningful increase in hemoglobin levels and maintained disease control in patients with PNH

FDA Approves Zevtera for Serious Bacterial Infections

The FDA has approved Basilea’s Zevtera (ceftobiprole medocaril sodium) to treat three different infections: adults with Staphylococcus aureus bloodstream infections, including those with right-sided infective endocarditis; adults with acute bacterial skin and skin structure infections; and adult and pediatric patients three months to less than 18 years old with community-acquired bacterial pneumonia.

Zevtera is a beta-lactam antibiotic for intravenous administration. It has activity against Gram-positive bacteria including methicillin-resistant strains of Staphylococcus aureus (MRSA). Zevtera will launch later this year with an unnamed U.S. partner, a Basilea spokesperson said. The partner will be announced by the middle of the year. Zevtera's price has not yet been determined.

For the majority of the uses (i.e., in the hospital), Zevtera is expected to be covered as part of the diagnosis-related group (DRG) bundled payment system. The DRG is determined by the diagnosis.

FDA Approves Fanapt for Bipolar 1

The FDA has approved Vanda Pharmaceuticals’ Fanapt (iloperidone) tablets to treat adults with acute manic or mixed episodes associated with bipolar I disorder. An estimated 2.8% of U.S. adults had bipolar disorder in the past year, according to the National Institute of Mental Health.

Fanapt is an atypical antipsychotic agent that has been used for the acute treatment of patients with schizophrenia since its FDA approval in 2009. For the full year 2023, Fanapt generated sales of $90.9 million, a decrease of 4% from 2022. The price for a supply of eight tablets is $261 according to Drugs.com. The company offers a $0 copay per month for those with commercial insurance.

The approval of Fanapt for use bipolar was based on a pivotal study in 414 patients who were randomized to received either Fanapt or placebo. The primary endpoint was assessed by the Young Mania Rating Scale (YMRS), a rating scale of clinical severity in the core symptoms of mania, at week four.

FDA Approves Abecma for Earlier Treatment of Multiple Myeloma

Abecma (idecabtagene vicleucel) is now approved to treat adults with relapsed and refractory multiple myeloma (R/R MM) after just two or more lines of therapy, including treatment with an immunomodulatory agent, a proteasome inhibitor and an anti-CD38 monoclonal antibody.

The approval is based on results from the phase 3 KarMMa-3 trial. In March 2024, the FDA Oncologic Drugs Advisory Committee (ODAC) voted 8-3 to recommend approval of Abecma based on the favorable benefit/risk profile for patients with triple-class exposed R/R MM

Abecma is a B-cell maturation antigen (BCMA)–directed CAR T-cell therapy that already was first approved in March 2021 to treat adults with R/R MM after 4 or more lines of therapy. It was the first cell-based gene therapy approved to treat adults with MM.

FDA Accepts Supplemental BLA for Bimzelx in Hidradenitis Suppurativa

The FDA has accepted the supplemental biologics license application (sBLA) for Bimzelx (bimekizumab-bkzx) 1 mL to treat adults with moderate-to-severe hidradenitis suppurativa. The FDA is considering a separate BLA for a 2 mL syringe.

Hidradenitis suppurativa is a condition that causes small, painful lumps to form under the skin. Developed by UCB, Bimzelx is a humanized IgG1 monoclonal antibody that designed to selectively inhibit two key cytokines that drive inflammation – interleukin 17A (IL-17A) and interleukin 17F (IL-17F). It is already available to treat moderate-to-severe plaque psoriasis. UCB launched Bimzelx in November 2023 for the plaque psoriasis indication as both an autoinjector and a pre-filled syringe. The list price is $7,200 per syringe.

FDA Sets Review Date for Datopotamab Deruxtecan in Breast Cancer

The FDA has set the Prescription Drug User Fee Act date for the first quarter of 2025 for a decision on datopotamab deruxtecan, which is being reviewed to treat adult patients with unresectable or metastatic hormone receptor (HR)-positive, HER2-negative breast cancer. If approved, it would be indicated for those who have received prior systemic therapy for unresectable or metastatic disease.

Datopotamab deruxtecan is a TROP2-directed antibody drug conjugate (ADC) being jointly developed by AstraZeneca and Daiichi Sankyo. TROP2 is overly expressed in breast cancer and plays a role in tumor growth and metastasis.

The biologics license application is based on results from the pivotal TROPION-Breast01 phase 3 trial. In the trial, datopotamab deruxtecan demonstrated a statistically significant and clinically meaningful improvement in progression-free survival (PFS) compared with chemotherapy, one of the primary end points. For second primary end point of overall survival (OS), interim results favored datopotamab deruxtecan over chemotherapy but were not mature at the time of data cutoff.

The trial found that datopotamab deruxtecan reduced the risk of disease progression or death by 37%, providing a two-month median progression-free survival benefit. Results also showed a confirmed objective response rate (ORR) of 36.4% in patients treated with datopotamab deruxtecan compared with an ORR of 22.9% with chemotherapy.

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