New Biotherapies Approved in the EU, UK and US

Health regulators in the European Union, United Kingdom and United States recently granted new approvals for biotherapies with indications for patients with cancer and rare diseases.

European Commission Expands Liso-Cel Approval to R/R MCL

The European Commission (EC) recently expanded approval of Bristol Myers Squibb’s lisocabtagene maraleucel (liso-cel, Breyanzi) to include adults with relapsed or refractory mantle cell lymphoma (MCL) who have received at least two prior lines of systemic therapy, including a BTK inhibitor.

The EC based the decision on the MCL cohort of the phase 1 TRANSCEND NHL 001 study, in which 82.7% of patients responded to therapy and 71.6% achieved a complete response. Safety findings were consistent with liso-cel’s established profile, with most cytokine release syndrome and neurologic events occurring within the first two weeks and resolving with standard management.

The authorization applies across all European Union and European Economic Area countries. It marks the fourth approved indication for liso-cel in Europe.

NHS England to Provide Obe-Cel for Adults With B-ALL

NHS England announced that the agency will provide obecabtagene autoleucel (obe-cel, Autolus Therapeutics) to adults aged 26 and older with relapsed or refractory B-cell acute lymphoblastic leukemia (B-ALL). The announcement follows approval from the National Institute for Health and Care Excellence. The agency estimates that approximately 50 patients per year in England will be eligible for treatment.

FDA Approves Second Novartis Gene Therapy for SMA

The U.S. Food and Drug Administration recently approved onasemnogene abeparvovec-brve (Itvisma, Novartis) for the treatment of spinal muscular atrophy (SMA) in adults and children aged 2 years and older with a confirmed SMN1 mutation. The treatment is an adeno-associated virus (AAV) vector-based gene therapy delivered as a single intrathecal injection.

The active ingredient in onasemnogene abeparvovec-brve is identical to that of onasemnogene abeparvovec-xioi (Zolgensma), Novartis’s first SMA gene therapy. However, onasemnogene abeparvovec-xioi is formulated at a different concentration and administered intravenously to patients younger than 2 years based on body weight. Onasemnogene abeparvovec-brve is instead delivered directly into the central nervous system at a fixed dose, allowing targeted delivery to motor neurons and expanding treatment options for older patients.