FDA Grants CYP-001 Orphan Drug Designation

This morning, the U.S. Food and Drug Administration (FDA) granted orphan drug designation to Cynata Therapuetics Limited (ASX:CYP) for CYP-001, the company’s potential treatment for acute graft versus host disease (GvHD).

The future of the drug bares promise as it embarks on its Phase 1 study, which was initiated in May 2017 with the hopes of reaching a primary completion date in June 2018. The study’s primary outcome is to evaluate the efficacy and safety of CYP-001.

While enrollment is ongoing, the study estimates to have 16 participants. Those aged 18 to 70 years who have a diagnosis using a consensus grading with steroid-resistant Grade II-IV acute GvHD after a haematopoietic stem cell transplant for a haematological disorder and a life expectancy of a minimum of 1 month are eligible to participate. Participants must also agree to have follow-up data collected 2 years following the initial dose of CYP-001.¹

GvHD occurs when a recipient’s donor bone marrow or stern cells attack.

The key data the study hopes to collect will include partial response (PR) and complete response (CR) rates and the overall survival of participants. Within the course of 100 days, patients will be evaluated at days 28 and 100 for treatment emergent adverse effects, physical examinations, safety laboratory evaluations, vital signs, and pulse oximetry.

The first 8 participants will receive a CYP-001 dose of 1 million cells per kg up to a maximum dose of 100 million cells on days 0 and 7, and they will be enrolled in Cohort A. The other 8 participants will be enrolled in Cohort B and will receive a dose of 2 million cells/kg, up to a maximum dose of 200 million cells, on days 0, 3, 7, 14, 21, 28, 60 and 100.¹ One hundred days after the first dose of CYP-001 is the primary evaluation period, while a long-term follow-up 2 years after the first dose concludes the study (March 2020).

CYP-001 is Cynta’s lead mesenchymal stem cell (MSC) product. Its orphan-drug designation is significant since it is eligible for key incentives, such as tax credits, FDA waivers, and an extended period of marketing exclusivity. It also positions itself for cost-effective commercialization in the U.S, which is the world’s largest healthcare market.

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Reference:

1. “FDA Grants Orphan Drug Designation to Cynata’s Lead Cymerus MSC Product, CYP-00” Globe Newswire. 28, Mar. 2018.

2. Kelly, Kilian, PhD. “A Study of CYP-001 for the Treatment of Steroid-Resistant Acute Graft Versus Host Disease.” National Institute of Health. 26 Feb. 2018.