Four medicines received positive opinions from the European Medicines Agency's (EMA) Committee for Medicinal Products for Human Use (CHMP) last week, expanding treatment options for cancer, rare tumors, bleeding disorders, and severe bacterial infections across pediatric and adult populations.

Pediatric Formulation for Neurofibroma

The CHMP backed a new formulation of Koselugo (selumetinib, AstraZeneca AB) in granule-in-capsule form (5 mg and 7.5 mg). This extends the indication to children aged 1 to < 7 years with symptomatic, inoperable plexiform neurofibromas (PN) associated with neurofibromatosis type 1 (NF1), as well as older patients with swallowing difficulties.

NF1 is a genetic disorder caused by mutations that overactivate the RAS pathway, leading to complex tumors that grow along nerves. Selumetinib, a selective MEK inhibitor, blocks MAPK/ERK signaling to reduce PN growth and can shrink tumors or delay symptom progression.

A phase 2 trial including 50 pediatric patients demonstrated that 34 patients (68%) had a confirmed partial response and 28 had a durable response. Study findings suggested improvements in pain, function, and quality of life, supporting the formulation's use and continued treatment into adulthood for responders.

New Option for Relapsed Lymphoma Treatment 

The CHMP recommended expanding Minjuvi (tafasitamab, Incyte Biosciences) to a new indication: adult patients with relapsed or refractory follicular lymphoma (FL) grade 1-3a after at least one prior line of systemic therapy, in combination with lenalidomide and rituximab.

In the phase 3 inMIND study, approximately 548 adults with relapsed or refractory FL grades 1-3a were randomized to receive lenalidomide + rituximab with or without tafasitamab, and the tafasitamab arm achieved a median progression-free survival of 22.4 months vs 13.9 months in the control arm (hazard ratio, 0.43; P < .0001).

Minjuvi was already approved in the EU for diffuse large B-cell lymphoma (DLBCL) — the recent CHMP opinion applies only to the FL indication, not DLBCL.

Bleeding Disorder Treatment

Veyvondi (vonicog alfa, Baxalta Innovations GmbH - a subsidiary of Takeda) received a positive opinion extending its indication to include the treatment of hemorrhage in children with von Willebrand disease (vWD) when desmopressin is ineffective or contraindicated. The existing adult indications — covering both treatment and prevention of bleeding in surgical settings — remain unchanged. Veyvondi should not be used for the treatment of hemophilia A.

vWD is the most common inherited bleeding disorder caused by a deficiency or dysfunction of von Willebrand factor (vWF), a protein essential for blood clotting. Vonicog alfa is a recombinant human von Willebrand factor (rVWF). It works by replacing the missing or defective vWF, thus helping blood to clot. 

Antibiotic Extended to Adolescents

Xerava (eravacycline, Paion Pharma GmbH) received a positive opinion extending its indication to adolescents aged ≥ 12 years weighing ≥ 50 kg, in addition to adults, for the treatment of complicated intra-abdominal infection (cIAI). Prescribers are advised to follow official antimicrobial stewardship guidance when using this agent.

cIAIs are severe, polymicrobial infections that extend into the peritoneal space and often involve resistant pathogens.

Eravacycline is a synthetic fluorocycline antibiotic that inhibits bacterial protein synthesis by binding to the 30S ribosomal subunit. It offers broad-spectrum activity against gram-positive, gram-negative, and anaerobic bacteria, including multidrug-resistant organisms such as ESBL-producing Enterobacteriaceae and some colistin-resistant strains. The IGNITE 1 and IGNITE 4 trials, which included adults, demonstrated that intravenous eravacycline is noninferior to carbapenems (ertapenem, meropenem) for clinical cure rates in cIAI, with a favorable tolerability profile.

Koselugo and Minjuvi both remain under additional monitoring, hold conditional marketing authorization, and retain their orphan medicine designation.

For all four newly approved product extensions, detailed recommendations for use will be given in the updated Summary of Product Characteristics to be published on the EMA website in all official European Union languages after marketing authorization has been granted by the European Commission.