The European Medicines Agency (EMA) Committee for Medicinal Products for Human Use (CHMP) has recommended eight new medicines for approval in its May meeting, including a new gene therapy.
Leading the recommendations is bluebird bio’s Skysona (elivaldogene autotemcel) which has been accepted for the treatment of early cerebral adrenoleukodystrophy (CALD).
The committee has recommended granting a marketing authorisation for the gene therapy for CALD patients without a matched sibling haematopoietic stem cell donor.
If approved by the European Commission, Skysona will become the first one-time gene therapy approved to treat CALD.
The CHMP has also adopted a positive opinion for Albireo’s Bylvay (odevixibat) for the treatment of progressive familial intrahepatic cholestasis (PFIC) in patients aged six months or older.
PFIC is a rare disease, mainly affecting young children, that causes progressive and life-threatening liver disease.
Rhythm Pharmaceuticals’ Imcivree (setmelanotide) has also received a positive CHMP opinion, for the treatment of obesity and the control of hunger associated with genetic deficiencies of the MC4R pathway.
Meanwhile, Almirall’s Klisyri (tirbanibulin mesylate) has been recommended for the field treatment of non-hyperkeratotic, non-hypertrophic actinic keratosis.
Bioprojet Pharma’s Ozawade (pitolisant) has also scored a positive opinion from the CHMP for the treatment of excessive daytime sleepiness in obstructive sleep apnoea.
Gedeon Richter’s Ryeqo (relugolix/estradiol/norethisterone acetate) and Bayer’s Verquvo’s (vericiguat) have been recommended for approval, for the treatment of symptoms of uterine fibroids and of symptomatic chronic heart failure in adults with reduced ejection fraction, respectively.
Lastly, the CHMP recommended granting a marketing authorisation for the generic medicine Icatibant Accord (icatibant) for the treatment of acute attacks of hereditary angioedema.