EMA recommends five new medicines addressing rare diseases and aggressive cancers

The European Medicines Agency (EMA) has recommended five new medicines for approval following its Committee for Medicinal Products for Human Use (CHMP) meeting in March 2026, marking another step forward in addressing unmet medical needs across oncology, rare diseases and paediatrics.

Among the highlights is Adstiladrin (nadofaragene firadenovec), recommended for adults with Bacillus Calmette-Guérin (BCG)-unresponsive non-muscle invasive bladder cancer. The committee also recommended Imdylltra (tarlatamab) for approval, a novel treatment for relapsed extensive-stage small cell lung cancer (SCLC), a disease with particularly poor prognosis and limited treatment options.

Commenting on Imdylltra, Amgen said: “SCLC is one of the most aggressive solid tumors, accounting for approximately 13-15% of lung cancer diagnoses worldwide. While patients may initially respond to chemotherapy, most relapse within months and long-term survival remains low. If approved, this would mark an important milestone for patients and clinicians across Europe and underscore Amgen’s continued progress in advancing innovative immuno-oncology therapies.”

Imdylltra falls under the scope of the EU Health Technology Assessment Regulation. The Member State Coordination Group on Health Technology Assessment (HTA) will endorse the draft joint clinical assessment for this product within 30 days of the European Commission granting marketing authorisation.

The CHMP also issued a positive opinion under exceptional circumstances for Joenja (leniolisib), developed by Pharming, for activated phosphoinositide 3-kinase delta syndrome (APDS), a rare and potentially life-threatening immune disorder affecting just one to two people per million globally.

Fabrice Chouraqui, CEO of Pharming, said: “For patients living with APDS, there remains a significant unmet medical need. By targeting the underlying cause of the disease, Joenja could mark a step-change in APDS care in Europe. Today’s positive CHMP opinion… reflects the strength of the clinical data and the dedication of the patients, families, and physicians who participated in the clinical studies.”

Another notable recommendation was for Zepzelca (lurbinectedin), developed by PharmaMar, as a maintenance treatment for extensive-stage SCLC. The decision is based on Phase 3 IMforte trial data showing a 46% reduction in disease progression or death and a 27% reduction in mortality when combined with atezolizumab.

Luis Mora, Managing Director of PharmaMar, commented: “The CHMP's positive opinion represents a very important milestone in facilitating access for European patients to a new therapeutic option. It also represents important recognition of our Company's commitment to research and development of innovative new compounds.”

Two additional recommendations include Bopediat (furosemide), a paediatric-use medicine for treating oedema and hypertension in children with chronic kidney disease, and an extension of Zepzelca’s orphan drug designation.

Beyond new approvals, the CHMP recommended 13 extensions of therapeutic indications for existing medicines and confirmed its refusal to extend the use of Hetlioz (tasimelteon) for Smith-Magenis syndrome. It also concluded a review requested by the European Commission, recommending that Tecovirimat SIGA should no longer be used to treat mpox.

The committee’s decisions now move to the European Commission for final approval, which typically follows within a few months.