Vertex and Alnylam orphan drugs set for EU approval

Orphan drugs for cystic fibrosis and the hereditary rare disease hATTR are on course for European approval after winning the backing of a key regulatory advisory committee.

The CHMP’s recommendations for Alnylam’s Onpattro (patisiran) and Vertex’s Symkevi (tezacaftor/ivacaftor) should translate into full EU approval within the next three months.

Alnylam’s Onpattro is a treatment for hereditary transthyretin-medicated amyloidosis (hATTR) in adults with stage 1 or stage 2 polyneuropathy. The rare and life-threatening disease is caused by mutations in the transthyretin (TTR) gene.

Onpattro’s active ingredient is made of a small strand of synthetic genetic material called ‘small interfering RNA’ (siRNA) that’s designed to attach to the genetic material of the cells responsible for producing the TTR protein and block its production in the liver.

Theresa Heggie, head of Europe at Alnylam Pharmaceuticals, said: “We are ready to launch patisiran following the EC decision, and hope that it will help to meet the pressing need for new treatment options for patients living with hATTR amyloidosis in Europe.”

Onpattro is set to be the second medicine approved for hATTR amyloidosis, following the European’s Commission’s authorisations of Akcea and Ionis’ Tegsedi (inotersen) earlier this month.

The CHMP also gave the green light to Symkevi, an extension of Vertex’s ivacaftor franchise of Kalydeco (ivacaftor) and Orkambi (ivacaftor+lumacaftor).

If approved Symkevi will be used in combination with Kalydeco and be the treatment for the CFTR protein defect in CF patients who have one copy of the F508del mutation and a copy of one of 14 mutations that result in residual CTFR activity.

Alongside Onpattro and Symkei the CHMP also recommended two paediatric licences for Kigabeq (vigabatrin), for the treatment of West's syndrome and resistant partial epilepsy, and childhood insomnia treatment Slenyto (melatonin).

It also backed Pierre Fabre's melanoma drugs Braftovi (encorafenib) and Mektovi (binimetinib), AstraZeneca's Imfinzi (durvalumab) in lung cancer and Lilly’s breast cancer drug Verzenios (abemaciclib).

There were further recommendations for two biosimilar versions of Amgen’s Neulasta (pegfilgrastim), with Accord Healthcare’s Pelgraz and ERA Consulting’s Udenyca backed for approval.

But the Committee voted against extending the use for BMS’ Opdivo (nivolumab) and Yervoy (ipilimumab) combination to a first line kidney cancer treatment and against Amgen’s Blincyto in patients with minimal residual disease after treatment for B-precursor acute lymphoblastic leukaemia.