EMA recommends bluebird bio's Skysona, seven other medicines

Regulatory NewsRegulatory News | 24 May 2021 |  By 

The European Medicines Agency (EMA) issued a favorable opinion for bluebird bio’s Skysona (elivaldogene autotemcel), a gene therapy that treats early cerebral adrenoleukodystrophy (CALD). Seven other medicines also received positive recommendations from the agency’s Committee for Medicinal Products for Human Use (CHMP) during its May meeting.
Skysona’s status as an advanced therapy medicinal product (ATMP) means it was first assessed by EMA’s Committee for Advanced Therapies (CAT); it also received orphan designation and was accepted into EMA’s PRIME scheme in 2018. Ongoing data collection to assess long-term efficacy and safety will be reported to EMA in post-marketing safety reports.
Albireo’s Bylvay (odexivixibat), another drug with orphan designation, received CHMP’s first positive recommendation for a medicine to treat progressive familial intrahepatic cholestasis. The committee’s recommendation was for a marketing authorization under exceptional circumstances after a development program that included acceptance into the PRIME scheme and an accelerated assessment.
A treatment for obesity and control of hunger in individuals with melanocortin 4 receptor pathway deficiencies also received a positive opinion: Rhythm Pharmaceuticals’ Imcivree (setmelanotide) was the third orphan-designated drug reviewed by CHMP in May.
Bayer’s Verquvo (vericiguat received a positive recommendation for treating chronic heart failure in adults with reduced ejection fraction. Ozawade (pitolisant), marketed by Bioprojet Pharma, was recommended to treat excessive daytime sleepiness in patients with obstructive sleep apnea.
Gedeon Richeter’s Ryeqo (relugolix/estradiol/norethisterone acetate) was recommended to treat symptomatic uterine fibroids in reproductive-age adult women, while Almirall received a positive opinion for its topical microtubule inhibitor Klisyri (tirbanibulin mesylate) to provide field treatment for certain forms of actinic keratoses.
Just one generic medicine, Accord’s icatibant, indicated to treat hereditary angioedema attacks, received a positive recommendation from CHMP.
At the May meeting, 17 medicines in total received recommendations for extension of indication, with two medicines, Libtayo and Darzalex, receiving recommendations for two indications each. No medicines received negative opinions in May; CHMP has not issued any negative opinions to date in 2021.
In COVID-19-related decisions, CHMP concluded that the monoclonal antibody (mAb) sotrovimab may be used to treat confirmed COVID-19 in those aged 12 years and up who are at risk of developing severe disease, but do not require supplemental oxygen. The decision was made after CHMP reviewed quality data and results of a planned interim analysis of use of the mAb in the indicated population.
EMA also issued more advice regarding the rare instances of thrombocytopenia, thrombosis, or both that can occur after receipt of the AstraZeneca COVID-19 vaccine, also known as Vaxzevria. CHMP concluded a review through the Type II Variation procedure at its May meeting, concluding that anyone who experience both low platelets and clotting after receiving the vaccine should not receive it again.
Individuals who develop clots within 3 weeks after receiving the vaccine should be checked for low blood platelets, and those with low platelets should be checked for thrombosis. Patients with the clotting-low platelet condition should receive specialist care.


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