Spherix Global Insights

June 08, 2022

Nephrologists Await Advanced Pipeline Agents, Including Venglustat (Sanofi), Lucerastat (Idorsia), and PRX-102 (Protalix), to Address High Unmet Need in Fabry Disease, Spherix Reports

Diagnosis, treatment, management, the increasing role of genetic testing, and the future promise of gene therapy captured in new report

EXTON, Pa., / June 8, 2022/PRNewswire – Fabry disease is an extremely rare genetic condition most prevalent in men and potentially underdiagnosed in women. Nephrologists rank it as one of the most difficult conditions to diagnosis and manage, placing it ahead of other extremely rare conditions such as Alport syndrome and ANCA vasculitis.

The newest report included in Spherix’s Market Dynamix™: Fabry Disease (US)  service, conducted in May 2022, evaluates the evolving treatment landscape and the potential paradigm shifts in diagnosis, treatment, and management that could have a significant impact on the future of patient care.

Insights collected from the report provide in-depth insights on a range of critical topics including screening, diagnosis, monitoring, patient management, and referral patterns across specialties. Feedback about current and future use of enzyme replacement treatments identified key considerations for potential increased use of the oral enzyme replacement therapy, Amicus’ Galafold, as well as expectations of the emerging pipeline and the potential of gene therapy to transform the treatment paradigm.

To understand perceptions and attitudes of future treatments, Spherix assessed physician awareness of pipeline products (aided and unaided) and reactions to summaries of several therapies, including likelihood to prescribe. Assets evaluated include:

  • Venglustat (Sanofi)
  • Lucerastat (Idorsia Pharmaceuticals)
  • PRX-102 (Protalix Biotherapeutics
  • ST-920 (Sangamo Therapeutics)

Looking ahead, nephrologists note the increasing use of genetic testing, as costs and access to the tests continue to improve, and the high value in Fabry disease as a diagnostic tool. Nephrologists pointed to the potential of gene therapy to transform the treatment landscape and deliver a significant advance if proven to be safe and effective.

About Market Dynamix™
Market Dynamix™ is an independent service providing analysis of markets anticipated to experience a paradigm shift within the next three to five years. Insights highlight market size, current treatment approaches, unmet needs, and expert opinions on the likely disruption introduced by pipeline agents.

Learn more about our services here.

About Spherix Global Insights
Spherix Global Insights is a hyper-focused market intelligence firm that leverages our own independent data and expertise to provide strategic guidance, so biopharma stakeholders make decisions with confidence. We specialize in select immunology, nephrology, and neurology markets.

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