Nine medicines recommended for approval, including two orphans

The European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) recommended nine medicines for approval, including two orphan medicines, at its May 2018 meeting.

The CHMP recommended granting a marketing authorisation for Aimovig (erenumab), the first human monoclonal antibody therapy for prevention of migraine. Aimovig belongs to a new class of medicines that work by blocking the activity of calcitonin gene-related peptide, a molecule that is involved in migraine attacks.

The Committee recommended granting a marketing authorisation for Tegsedi (inotersen), a medicine for patients with hereditary transthyretin amyloidosis that aims to affect the course of the disease and improve quality of life. This medicine was reviewed under EMA’s accelerated assessment mechanism, reserved for medicines of major public health interest. Tegsedi was designated as an orphan medicine during its development.

Myalepta (metreleptin) received a positive opinion for the treatment of leptin deficiency. Myalepta was designated as an orphan medicine during its development.

The CHMP recommended granting a marketing authorisation for Rxulti (brexpiprazole), for the treatment of schizophrenia.

Four biosimilar medicines received a positive opinion from the Committee: HalimatozHefiya and Hyrimoz, all containing adalimumab, were recommended for the treatment of certain inflammatory and autoimmune disorders; and Trazimera(trastuzumab), was recommended for the treatment of breast and gastric cancer.

The Committee recommended for approval the generic medicine Nityr (nitisinone), for the treatment of hereditary tyrosinemia type 1.

Negative opinion on new medicine

The CHMP adopted a negative opinion refusing a marketing authorisation for Exondys(eteplirsen). Exondys was expected to be used to treat Duchenne muscular dystrophy. The applicant requested a re-examination of this negative opinion on 1 June 2018.

Update on re-examination

The applicant for Alsitek (masitinib) has withdrawn the request for re-examination of the Committee’s negative opinion for this medicine. For more information please see the question-and-answer document.

Three recommendations on extensions of therapeutic indication

The Committee recommended extensions of indications for BriviactTranslarna and Xeljanz.

Start of referral: metamizole-containing medicines

The Committee also started a review of medicines containing the painkiller metamizole. There are substantial differences between member states in the recommended maximum daily doses of the medicine and the contraindications on its use during pregnancy or in women who are breast feeding.

Outcome of review on Esmya

The CHMP recommended several measures to minimise the risk of rare but serious liver injury with Esmya (ulipristal acetate), for the treatment of moderate to severe symptoms of uterine fibroids.

Outcome of review on Keytruda and Tecentriq

The CHMP recommended restricting the use of Keytruda (pembrolizumab) and Tecentriq (atezolizumab) as first line-treatments for urothelial cancer, because new data indicate that these medicines may not work as well as chemotherapy in some patients with low levels of the protein PD-L1.

Withdrawal of application

The application for an initial marketing authorisation for Restaysis (ciclosporin) was withdrawn. This medicine was intended to be used for the treatment of moderate dry eye disease.

Posted on the EMA website on 1 June 2018