Cell/ gene therapy industry 2016 – expectations for market approvals

by Alexey Bersenev on January 21, 2016 · 2 comments

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Last 3 years were not very successful for approvals of cell/ gene therapy products on the market. From zero (in 2014) to three (in 2013 and 2015) product approvals per year – very typical for the industry. I hope, this year will be a beginning of the change. In this post I’m trying to summarize information about potential approvals for 2016 – beginning of 2017. Please let me know if I missed anything or was too optimistic in prognosis.

  1. MACI approval in US. Vericel just submitted BLA to FDA for approval of MACI – autologous chondrocytes on the matrix. MACI was approved in Europe by EMA in 2013, but was suspended soon after. Sanofi divested MACI to Vericel in 2014.
  2. GSK with gene therapy of ADA-SCID. In May of 2015 GSK filed application to EMA for approval of gene therapy drug (CD34+ auto- cells modified with ADA) in inherited immunodeficiency (“bubble boy disease” – ADA-SCID). This application is a results of 5-years collaboration with Italian San Raffaele Telethon Institute (TIGET). Because this is a rare disease, GSK submitted results for experimental treatment of 18(!) patients.
  3. Spark Therapeutics with gene therapy of eye disease. After successful results of Phase 3 trial, US-based company Spark Therapeutics is hoping to get FDA approval for the first commercial gene therapy product in US by the beginning of 2017. Spark will file BLA in the second half of this year.
  4. Heartcel approval in UK by beginning of 2017. This year, UK-based company Cell Therapy Ltd is planning to submit application to EMA for their cardiac cell therapy product Heartcel. Company is planning to obtain conditional approval in 2017.
    Stempeucel and Stempeutron launch in India. Stempeutics Research is hoping to get regulatory approval for adipose tissue processing device Stempeutron by the middle of this year in India. Company also submitted application for marketing authorization of mesenchymal stem cell-based drug Stempeucel for critical limb ischemia.
  5. Tigenix stem cell product for Crohn’s disease fistulas. Belgian company Tigenix reported results of Phase 3 results last year and planning to file for market authorization by EMA of their adipose stem cell-based product this year.
  6. Approvals in S. Korea. I’m expecting approval of cell therapy product Cerecellgram for stroke by Korean company FCB-Pharmicell. I’m also expecting to hear some good news from Medipost. Their product Pneumostem could get early market access in Korea for orphan indication.
  7. First CAR T-cell approvals. My expectation for the first CAR T-cell approval is beginning of 2017. I think, it will be Novartis with CD19-CART for pediatric ALL. Kite Pharma may get approval in US for the same indication few months later.

Finally, I feel like some surprises will come from China and Japan. I’m not sure what products, but something…

{ 2 comments… read them below or add one }

Dean Krauss January 21, 2016 at 12:39 am

How about BLUE B-thal approval in europe?


Matt February 11, 2016 at 5:26 pm

If a sponsor completes trials for (say) cell therapy in knee OA, would they have to complete the trial process again for the body’s hundred other joints?


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