The global hemophilia gene therapy market, by Hemophilia Type (Hemophilia A and Hemophilia B), and by Region (North America, Latin America, Europe, Asia Pacific, Middle East, and Africa), was valued at US$ 00.0 million in 2017 and is projected to exhibit a CAGR of 56.0% over the forecast period (2018 – 2026).
Hemophilia is one of the prominent genetic disorders that is characterized by the absence of clotting factors of specific type resulting in lack of coagulation after blood loss. This may result in serious health complications. Bleeding may be due to injury to external tissue or spontaneous bleeding in muscles and joints. Existing therapy offers symptomatic approach where clotting factors that are absent are provided externally on regular basis. However, there are many limitations of this approach such as toxicity, short term efficacy, high cost, and accessibility. Gene therapy offers solutions to all these limitations. Most important is the ability of the hemophilia gene therapy to address the root cause of hemophilia, which is caused due to genetic mutations of the gene that codes for the clotting factors VIII (Hemophilia A) and clotting factors IX (Hemophilia B). In gene therapy, specific gene is inserted into viral vectors (rAAV is most commonly used as vectors) and this vector-based gene is injected intravenously into hemophilic patients, which then codes for formation of coagulation factors.
Growing patient base for hemophilia Increasing prevalence of hemophilia worldwide is expected to drive growth of the hemophilia gene therapy market. In 2016, World Federation of Hemophilia stated that 1 in 5,000 people are affected by hemophilia A and 1 in 50,000 are affected by hemophilia B. According to the National Hemophilia Foundation factsheet 2018, over 400,000 individuals were affected by hemophilia worldwide. Furthermore, according to same source, an estimated 75% of patients suffering from hemophilia have no access to treatment or receive inadequate treatment. According to the Centers for Disease Control and Prevention (CDC), the median age for diagnosis is one month for individuals with severe symptoms, 8 months for individuals with moderate hemophilia, and 36 months for people with mild hemophilia.
Strong product pipeline for hemophilia gene therapy There are numerous pharma and biotech companies that are engaged in developing hemophilia gene therapy. Most of the companies are targeting hemophilia A, which has approximately 80% share in overall hemophilia patients. BioMarin Pharmaceuticals’ investigational gene therapy, Valoctocogene roxaparvovec for treating hemophilia A, is currently in phase III and Sangamo therapeutics’ investigational gene therapy SB – 525 is in phase I/II, which gave positive preliminary data in August 2018. In December 2018, Spark Therapeutics announced updated preliminary data for the ongoing phase 1/2 clinical trial of investigational SPK-8011 in hemophilia A. Shire PLC’s gene therapy SHP654 for hemophilia A is currently in phase I of clinical development. It is expected that the first gene therapy would enter the market by 2022. Overall, as per database of clinicaltrial.gov, there are around 17 investigational gene therapies under development. Although hemophilia gene therapy may be accessible to only limited number of patients initially due to uniqueness of therapy, it may be widely used over the period, majorly in emerging economies in Asia Pacific and the Middle East, where coagulation factors replacement therapy has limited access.
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There are various steps taken by the U.S. FDA to speed up the clinical development of hemophilia gene therapy. The U.S. FDA has allotted breakthrough therapy designation to several investigational gene therapy. For instance, in 2017, the U.S. Food and Drug Administration granted breakthrough therapy designation to valoctocogene roxaparvovec of BioMarin Pharmaceuticals. Inc. Earlier in 2016, company received orphan drug designation for valoctocogene roxaparvovec. The U.S FDA also announced new draft guidelines in 2018 that provides information about the required design and other parameters for an accelerated approval approach, which would help the developers of human gene therapy (GT) products for the treatment of hemophilia A & B.
Key Takeaways of the Hemophilia Gene Therapy Market:
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