The Committee for Medicinal Products (CHMP) of the European Medicines Agency (EMA) decided to approve five new drugs to treat diseases that show no improvement with existing formulations at its last meeting in March.

More specifically, the Committee recommended the conditional granting of a marketing authorisation for Adstiladrin (nadofaragene firadenovec), for the treatment of adult patients with non-myosinophilic bladder cancer unresponsive to standard therapy, with carcinoma in situ with or without papillary tumours.

The CHMP recommended granting marketing authorization for Imdylltra (tarlatamab), a new treatment for relapsed extensive-stage small cell lung cancer, which addresses an unmet medical need in adults with a poor prognosis and limited treatment options.

Also, Joenja (leniolisib) received a positive opinion for marketing authorization under exceptional circumstances for the treatment of activated phosphoinositide 3-kinase delta syndrome (APDS) in adults and adolescents aged 12 years and older and weighing 45 kg or more. APDS is a rare, inherited, progressive, and potentially life-threatening immune system disease. It is worth noting that the incidence of the disease worldwide is estimated at 1 to 2 people per million.

A positive opinion was also adopted for Zepzelca (lurbinectedin) for maintenance therapy in patients with advanced stage small cell lung cancer whose disease has not progressed after initial induction therapy.

At the same time, the CHMP recommended granting a Paediatric Use Marketing Authorisation (PUMA) for Bopediat (furosemide) for the treatment of oedema (swelling due to excess fluid) of cardiac or renal origin, oedema of hepatic origin and hypertension in children from birth to under 18 years of age with chronic kidney disease. The medicine was submitted as a hybrid application, based partly on preclinical trial results and clinical studies of an already approved reference medicine and partly on new data.

Recommendations for extensions of therapeutic indications for 13 medicines

The EMA’s Pharmaceutical Committee recommended extensions of indications for 13 medicines already authorised in the EU:Besponsa, Capvaxive, Feraccru, Hetronifly (two extensions of indications), Hympavzi, Imcivree, Lojuxta, Mekinist (two extensions of indications), mResvia, Namuscla, Retsevmo, Sotyktu and Tafinlar (two extensions of indications).

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After reconsidering its initial opinion, the committee affirmed its recommendation to deny a change to the marketing authorization for Hetlioz (tasimelteon). The change was to expand the indication to include the treatment of nighttime sleep disorders in adults and children aged 3 to 15 years with Smith-Magenis syndrome, a rare inherited disorder characterized by developmental delay, behavioral problems and sleep disturbances.

Antiviral drug for mpox

During the same meeting, the Committee completed its evaluation of Tecovirimat SIGA (tecovirimat), an antiviral drug that had been approved for the treatment of smallpox, mpox and cowpox, three infections caused by viruses of the same family (orthopoxviruses). The CHMP recommended that Tecovirimat SIGA should no longer be used to treat mpox.

In addition, the Committee approved a new route of administration, subcutaneous, together with a new pharmacotechnical form and new content for Sarclisa, an anti-cancer medicine used to treat adults with multiple myeloma.