The European Medicines Agency's (EMA) Committee for Medicinal Products for Human Use (CHMP) has recommended nine medicines for approval, including first two CAR-T cell therapies and six orphan medicines, at its June 2018 meeting14.

A) The nine medicines recommended for approval are:

1. Cablivi (caplacizumab) – for treatment of acquired thrombotic thrombocytopenic purpura (aTTP)

On June 28, 2018, the Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion, recommending the granting of a marketing authorisation for Cablivi, indicated for the treatment of adults experiencing an episode of acquired thrombotic thrombocytopenic purpura (aTTP), in conjunction with plasma exchange and immunosuppression15. Cablivi was designated as an orphan medicinal product on April 30, 2009.

Cablivi will be available as a 10 mg powder and solvent for solution for injection. The active substance in Cablivi is caplacizumab, a humanised bivalent nanobody that inhibits the interaction between von Willebrand factor and platelets (ATC code: B01AX07). As a result, caplacizumab prevents von Willebrand factor-mediated platelet adhesion, which is characteristic of aTTP. It also affects the disposition of von Willebrand factor, leading to transient reductions of total von Willebrand factor antigen levels and to concomitant reduction of factor VIII: C levels during treatment.

The applicant for Cablivi is Ablynx NV.

2. Duzallo (lesinurad / allopurinol) - for treatment of hyperuricaemia in gout patients

On June 28, 2018, the Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion, recommending the granting of a marketing authorisation for Duzallo, indicated in adults for the treatment of hyperuricaemia in gout patients who have not achieved target serum uric acid levels with an adequate dose of allopurinol alone16.

Duzallo is a fixed dose combination of two active substances, lesinurad and allopurinol. It will be available as filmcoated tablets (300 mg/200 mg and 200 mg/200 mg). Lesinurad is a selective uric acid reabsorption inhibitor that inhibits uric acid transporter 1, and allopurinol reduces uric acid production by inhibition of xanthine oxidase.

The applicant for Duzallo is Gruenenthal GmbH.

3. Kymriah (tisagenlecleucel) – for treatment of acute lymphoblastic leukaemia (ALL) and diffuse large B-cell lymphoma (DLBCL)

On June 28, 2018, the Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion, recommending the granting of a marketing authorisation for chimeric antigen receptors (CAR) T-cells medicine Kymriah. It is indicated for the paediatric treatment and for treatment of young adult patients (up to 25 years of age) with B-cell ALL that is refractory or in second or later relapse, and in adult patients with relapsed or refractory DLBCL after two or more lines of systemic therapy17. Kymriah is also one of the first medicines supported through EMA's PRIority MEdicines (PRIME) scheme to receive positive opinions from the CHMP. It was granted PRIME eligibility on June 23, 2017.

Kymriah will be available as a dispersion for infusion. The active substance of Kymriah is tisagenlecleucel, an autologous, immunocellular cancer therapy which involves reprogramming a patient's own T-cells to identify and eliminate CD19-expressing cells. This is achieved by addition of a transgene encoding a chimeric antigen receptor (CAR).

The applicant for Kymriah is Novartis Europharm Limited.

Download >> European Medicines Agency (EMA): Recommends Approval Of Nine Medicines In Its June Meeting

Footnotes

14 http://www.ema.europa.eu/ema/index.jsp?curl=pages/news_and_events/news/2018/06/news_detail_002981.jsp&mid=WC0b01ac058004d5c1

15 http://www.ema.europa.eu/docs/en_GB/document_library/Summary_of_opinion_-Initial_authorisation/human/004426/WC500251160.pdf

16 http://www.ema.europa.eu/docs/en_GB/document_library/Summary_of_opinion_-Initial_authorisation/human/004412/WC500251152.pdf

17 http://www.ema.europa.eu/docs/en_GB/document_library/Summary_of_opinion_-Initial_authorisation/human/004090/WC500251211.pdf  

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