multiple myeloma orphan drugs future (kev-shine http://www.flickr.com/photos/kevinshine/)

Orphan drugs have a future in multiple myeloma

In Clinical Development by Tim PeplowLeave a Comment

The recent late-stage success of Novartis’ orphan drug panobinostat for multiple myeloma has turned heads towards the large and exciting pipeline of experimental drugs for the rare bone marrow cancer.

Panobinostat (LBH589) blocks pan-deacetylase (pan-DAC), a key enzyme found in cancer cells. It is one drug in a class of drugs known as histone deactylase (HDAC) inhibitors, a class that also includes Merck’s Zolinza (vorinostat) and Acetylon Pharmaceutical’s rocilinostat (ACY-1215). Positive interim Phase Ib data of ricolinostat in MM were recently presented, with the drug being used alongside Velcade and dexamethasone, and Revlimid and dexamethasone. HDAC inhibitors slow cell division and promote apoptosis.

Another class of drug for MM is the proteasome inhibitors, which encompasses Millenium Pharmaceutical’s Velcade (bortezomib) and Onyx Pharmaceutical’s Kyprolis (carfilzomib). A proteasome inhibitor still in the experimental phase is ixazomib, developed by Velcade manufacturers Millenium.

Immunomodulatory drugs can also be used to tackle MM, working by encouraging the immune system to destroy the myeloma cells. This class includes Celgene’s Revlimid (lenalidomide) and Pomalyst (pomalidomide).

The fourth class of drugs for MM is the monoclonal antibodies, which include the promising elotuzumab from Bristol-Myers Squibb and AbbVie, Genmab’s daratumumab and Biotest’s BT-062.

You can read more about these treatments for multiple myeloma at The Myeloma Beacon >

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