February EMA/CHMP meeting results in seven positive new drug recommendations

At its last meeting in London before moving to new headquarters in Amsterdam as a result of Brexit, the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) made positive recommendations for the approval of seven novel medicines.

The Committee recommended granting a conditional marketing authorization for Ondexxya (andexanet alfa), from USA-based Portola Pharmaceuticals (Nasdaq: PTLA). This medicine is to be used as an antidote for adult patients taking the anticoagulant medicines apixaban or rivaroxaban, when reversal of their action is needed due to life-threatening or uncontrolled bleeding.

The Committee adopted a positive opinion for Palynziq (pegvaliase), from BioMarin Pharmaceutical (Nasdaq: BMRN), a new medicine for patients aged 16 and older with phenylketonuria, a rare but potentially serious inherited metabolic disease. Palynziq was designated as an orphan medicine during its development.

The CHMP recommended granting a conditional marketing authorization for Akcea Therapeutics’ (Nasdaq: AKCA) Waylivra (volanesorsen), the first medicine for the treatment of familial chylomicronemia syndrome, a rare genetic disease that prevents the body from breaking down fats. Waylivra was designated as an orphan medicine during its development. There is currently no authorized medicine available to treat this rare disease.

Zynquista (sotagliflozin), from Sanofi (Euronext: SAN), intended as an adjunct to insulin for certain patients with type 1 diabetes, received a positive opinion from the CHMP. Zynquista is indicated as an adjunct to insulin therapy to improve glycemic control in adults with type 1 diabetes mellitus who have failed to achieve adequate glycemic control despite optimal insulin therapy. Patients considered for this treatment should fulfil certain requirements and should have a body mass index (BMI) higher than 27 kg/m 2 .

The Committee recommended granting a marketing authorization under exceptional circumstances for GlaxoSmithKline’s (LSE: GSK) Dectova (zanamivir), for the treatment of complicated and potentially life-threatening influenza A or B virus infection in adult and pediatric patients (aged ≥6 months) when the patient’s influenza virus is known or suspected to be resistant to anti-influenza agents other than zanamivir, and/or other anti-viral agents for treatment of influenza, including inhaled zanamivir (Relenza), are not suitable for the individual patient..

The CHMP adopted a positive opinion, recommending the granting of a conditional marketing authorisation for Pfizer’s (NYSE: PFE) Lorviqua (lorlatinib), for the treatment of patients with anaplastic lymphoma kinase-positive advanced non-small cell lung cancer.

Skyrizi (risankizumab), from AbbVie (NYSE: ABBV), received a positive opinion for the treatment of moderate to severe psoriasis.