Eight new medicines recommended for approval, including one gene therapy medicine

EMA’s human medicines committee (CHMP) recommended eight medicines for approval at its May 2021 meeting.

The Committee recommended granting a marketing authorisation for Skysona* (elivaldogene autotemcel) for the treatment of early cerebral adrenoleukodystrophy (CALD) for patients without a matched sibling haematopoietic stem cell donor. As Skysona is a gene therapy, the CHMP’s positive opinion is based on an assessment by EMA’s Committee for Advanced Therapies (CAT). See more details in the news announcement in the grid below.

The Committee adopted a positive opinion, recommending the granting of a marketing authorisation under exceptional circumstances for Bylvay* (odevixibat) for the treatment of progressive familial intrahepatic cholestasis (PFIC) in patients aged 6 months or older. See more details in the news announcement in the grid below.

Imcivree* (setmelanotide) was granted a positive opinion by the Committee for the treatment of obesity and the control of hunger associated with genetic deficiencies of the melanocortin 4 receptor (MC4R) pathway. See more details in the news announcement in the grid below.

Klisyri (tirbanibulin mesylate) was granted a positive opinion for the field treatment of non-hyperkeratotic, non-hypertrophic actinic keratosis.

The CHMP adopted a positive opinion for Ozawade (pitolisant) for the treatment of excessive daytime sleepiness in obstructive sleep apnoea.

The Committee adopted a positive opinion for Ryeqo (relugolix / estradiol / norethisterone acetate) for the treatment of symptoms of uterine fibroids in adult women of reproductive age.

Verquvo (vericiguat) was granted a positive opinion by the Committee for the treatment of symptomatic chronic heart failure in adult patients with reduced ejection fraction.

The CHMP recommended granting a marketing authorisation for one generic medicineIcatibant Accord (icatibant) for the treatment of acute attacks of hereditary angioedema.

Seventeen recommendations on extensions of therapeutic indication

The Committee recommended extensions of indication for Blincyto, Eucreas, Evotaz, Galvus, Icandra, Jalra, Jardiance, Keytruda, Opdivo, Spherox, Xiliarx, Yervoy and Zomarist. The CHMP recommended two extensions of therapeutic indications for Libtayo and Darzalex.

COVID-19: Vaxzevria: further advice on blood clots and low blood platelets

The CHMP provided additional advice on blood clots or low blood platelets occurring after vaccination with Vaxzevria (formerly COVID-19 Vaccine AstraZeneca). See more details in the public health communication in the grid below.

COVID-19: Conditional marketing authorisation for Veklury renewed

The CHMP recommended to renew the conditional marketing authorisation for Veklury (remdesivir), the only authorised antiviral treatment for COVID-19. The medicine was first recommended for approval on 25 June 2020. It is authorised for use in patients (12 years and older) with pneumonia requiring supplemental oxygen (low- or high-flow oxygen or other non-invasive ventilation at the start of treatment).

The authorisation was granted on the condition that the company submits additional information on the quality, efficacy and safety of the medicine according to a timetable agreed with the CHMP. Following the assessment of the new information, the Committee considered that the benefits of Veklury continue to outweigh its risks and that the conditional authorisation should be renewed. Some data are still outstanding and need to be submitted by the end of the year for the next scheduled renewal.

Conditional marketing authorisation allows the approval of medicines that fulfil an unmet medical need, including during public health emergencies such as COVID-19, with less complete data than normally expected, if the benefit of a medicine’s immediate availability to patients outweighs the risk inherent to the fact that not all the data are yet available. This type of authorisation requires a re-assessment at least once per year to confirm that the balance of benefits and risks remains positive.

COVID-19: Advice on use of sotrovimab (VIR-7831) for treating COVID-19

The Committee completed its review on the use of the monoclonal antibody sotrovimab (also known as VIR-7831) to treat patients with COVID-19. This review was undertaken to provide a harmonised scientific opinion at EU level to support national decision-making on the possible use of the antibody prior to marketing authorisation. For more information, please see the news announcement in the grid below.

Confirmation of recommended use of Tecentriq

The Committee confirmed its previous recommendation to use Tecentriq (atezolizumab) only in combination with nab-paclitaxel and not with conventional paclitaxel when treating patients with locally advanced or metastatic triple-negative breast cancer that cannot be surgically removed. See more details in the public health communication in the grid below.

Posted on the EMA website on 21 May 2021