CMPH Recommends List of New Drugs and Therapies (Rare Disease, Gene Therapy and others)


The Committee for Medicinal Products for Human Use (CMPH) is a committee of the European Medicines Agency (EMA) that frames opinions regarding all medicines meant for human use. CMPH recently gave green signal to the following drugs and therapies as a marketing approval recommendations to treat different diseases.


Nature: Oral Drug

Company: Amicus Therapeutics Inc.

Disease: Fabry Disease

GALAFOLD MIGALASTAT of Amicus therapeutics recommended by CMPHAmicus disclosed CMPH approval for its Fabry disease therapy. Galafold migalastat is alpha galactosidase A activity enhancing small molecule. Its final approval from the European Commission of Medicine (ECM) is expected in the second half of this year. If approved by the ECM, Galafold migalastat shall be the first ever oral treatment for Fabry disease in Europe. Fabry disease is a genetic disorder caused by the abnormal metabolism of sphingolipids, the buildup of which causes the symptoms.


Nature: Gene Therapy

Company: GlaxoSmithKline plc

Disease: Severe Combined Immunodeficiency due to Adenosine Deaminase Deficiency (ADA-SCID)

GlaxoSmithKline plc Strimvelis recommended by CMPHGSK announced that the CMPH in collaboration with the Committee for Advances Therapies (CAT) has recently shown positive opinion and recommended the therapy GSK2696273 (Strimvelis) for marketing to treat patients with severe combined immunodeficiency due to adenosine deaminase deficiency.

This gene therapy was originally prepared by San Raffaele Telethon Institute for Gene Therapy (HSR TIGET, a collaboration of Ospedale San Raffaele and Fondazione Telethon). The therapy was then taken forward by GSK. The medicine, on approval by European Commission of Medicine, shall be used to treat patients with the SCID for whom no stem cell donor is available.


Nature:  Oral Drug

Company: Samsung Bioepis Co. Ltd

Disease: Rheumatoid arthritis, Crohn’s disease, ulcerative colitis, ankylosing spondylitis, psoriative arthritis and psoriasis

Flixabi from samsung bioepis got CPMH recommendationsFlixabi is the second anti-TNF (tumor necrosis factor) alpha biosimilar developed by Samsung Bioepis that has received positive recommendation for ECM approval. TNF is a cytokine, a cell signaling protein. TNF promotes the inflammatory responses which in turn cause clinical symptoms associated with autoimmune diseases such as mentioned above.


Nature: Oral Drug

Company: Johnson and Johnson

Disease: Relapsed and refractory multiple myeloma

Johnson and Johnson Darzalex got a positive opinion from CMPH

Johnson and Johnson’s Darzalex got a positive opinion on an application to have it as a new treatment for multiple myeloma in Europe. Johnson and Johnson said that it is very rare for the ECM to decide not to approve a drug recommended by the CMPH thus they are optimistic about its approval. Use of Darzalex shall be a single agent in adults with relapsed and refractory multiple myeloma who had been previously treated with a proteasome inhibitor and an immunomodulatory agent, and who experienced disease progression in their last therapy.


Nature: Injectable drug

Company: Bristol Mayer Squibb

Disease: Advanced melanoma

Bristol-Myers SquibbOpdivo received nod from the CMPH. It is already approved by the US Food and Drug Association (FDA) in USA. It is a cancer treatment drug used to treat skin cancers that cannot be treated by surgeries and have spread to other body parts. Opdivo operates by interfering with the growth and spread of tumor cells in the body. Opdivo has been approved by the FDA on an accelerated basis and it expects the same from ECM.

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