Tuesday, October 12 2021

Posted on May 24, 2021 | Through Kari oakes

The European Medicines Agency (EMA) has issued a favorable opinion for Skysona (elivaldogene autotemcel) from bluebird bio, a gene therapy that treats early cerebral adrenoleukodystrophy (CALD). Seven other drugs also received positive recommendations from the agency’s Committee for Medicinal Products for Human Use (CHMP) at its May meeting.

Skysona’s status as an advanced therapy medicine (ATM) means that it was first evaluated by the EMA’s Committee for Advanced Therapies (CAT); he also received orphan designation and was accepted into EMA’s PRIME program in 2018. Ongoing data collection to assess long-term efficacy and safety will be reported to EMA in reports post-market safety.

Bylvay (odexivixibat) from Albireo, another designated orphan drug, received the first positive recommendation from the CHMP for a drug to treat progressive familial intrahepatic cholestasis. The commission’s recommendation was for a marketing authorization in exceptional circumstances after a development program including acceptance into the PRIME scheme and an accelerated assessment.

Treatment for obesity and hunger control in people with deficiencies in the melanocortin 4 receptor pathway also received a positive review: Imciivree (setmelanotide) from Rhythm Pharmaceuticals was the third designated orphan drug reviewed by the CHMP in May.

Bayer’s Verquvo (vericiguat has received a positive recommendation for the treatment of chronic heart failure in adults with reduced ejection fraction. Ozawade (pitolisant), marketed by Bioprojet Pharma, has been recommended for the treatment of somnolence. excessive daytime in patients with obstructive sleep apnea.

Gedeon Richeter’s Ryeqo (relugolix / estradiol / norethisterone acetate) has been recommended to treat symptomatic uterine fibroids in adult women of childbearing age, while Almirall has received positive reviews for its topical microtubule inhibitor Klisyri (mesylate tirbanibulin) to provide field treatment for certain forms of actinic keratosis.

Only one generic medicine, Accord’s icatibant, indicated to treat attacks of hereditary angioedema, has received a positive recommendation from the CHMP.

At the May meeting, a total of 17 drugs received indication extension recommendations, with two drugs, Libtayo and Darzalex, receiving recommendations for two indications each. No drug received negative reviews in May; The CHMP has not issued any negative opinions to date in 2021.

In decisions related to COVID-19, the CHMP concluded that the monoclonal antibody (mAb) sotrovimab can be used to treat confirmed COVID-19 in people aged 12 years and older who are at risk of developing serious illness, but do not need additional oxygen. The decision was taken after the CHMP reviewed the quality data and the results of a planned interim analysis of the use of mAb in the indicated population.

The EMA has also issued more advice regarding rare cases of thrombocytopenia, thrombosis, or both that may occur after receiving the AstraZeneca COVID-19 vaccine, also known as Vaxzevria. The CHMP concluded a review of the type II variation procedure at its May meeting, concluding that anyone with both low platelet count and clotting after receiving the vaccine should no longer receive it.

Individuals who develop clots within 3 weeks of receiving the vaccine should be checked for low blood platelet count, and those with low platelet count should be checked for thrombosis. Patients with poor platelet coagulation should receive specialist care.


© 2021 Society of Regulatory Affairs Professionals.

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