Precigen announced the US Food and Drug Administration (FDA) has granted orphan drug designation (ODD) to PRGN-3006, a first-in-class investigational therapy using Precigen's non-viral UltraCAR-T™ therapeutic platform for patients with relapsed or refractory acute myeloid leukemia (AML). Precigen announced in Q3 2019 that it had completed enrollment for the first cohort of this clinical trial and expects an initial data readout in the second half of 2020.
The FDA grants ODD status to medicines intended for the treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the US. Medicines that receive the ODD designation may qualify for a number of incentives that help to expedite and reduce the cost of development, approval and commercialization.
"This regulatory designation underscores the critical medical need for new therapies to treat AML patients. AML is a progressive, debilitating and often fatal disease with limited treatment options," said Helen Sabzevari, PhD, President and CEO of Precigen*. "As the first regulatory designation for our proprietary UltraCAR-T platform, this orphan drug designation helps to advance the PRGN-3006 investigational therapy and provides important incentives and support to deliver this medicine as rapidly as possible for those patients suffering from this disease."
Subscribe to our e-Newsletters
Stay up to date with the latest news, articles, and events. Plus, get special offers
from American Pharmaceutical Review – all delivered right to your inbox! Sign up now!
PRGN-3006 utilizes Precigen's transformative UltraCAR-T therapeutic platform, which eliminates ex vivo expansion, reduces manufacturing time and provides the ability to administer CAR-T therapy to patients only one day after non-viral gene transfer at the cancer center. PRGN-3006 UltraCAR-T is a multigenic CAR-T cell treatment utilizing Precigen's advanced non-viral gene delivery system to co-express a chimeric antigen receptor, membrane-bound interleukin‐15 (mbIL15), and a kill switch for better precision and control in targeting relapsed or refractory AML and higher risk MDS.
AML is a cancer that starts in the bone marrow, but most often moves into the blood1. Though considered rare, AML is among the most common types of leukemia in adults2. In 2019, it was estimated that 21,450 new cases of AML would be diagnosed in the US2. AML is uncommon before the age of 45 and the average age of diagnosis is about 682. The prognosis for patients with AML is poor with an average 5‐year survival rate of approximately 25 percent overall, and less than a 5 percent 5‐year survival rate for patients older than 653. Amongst elderly AML patients (≥ 65 years of age), median survival is short, ranging from 3.5 months for patients 65 to 74 years of age to 1.4 months for patients ≥ 85 years of age 3.
FDA orphan drug designation is granted to drugs intended to treat rare diseases or disorders that affect fewer than 200,000 people in the US. Medicines that receive the ODD designation may qualify for a number of incentives that help to expedite and reduce the cost of development, approval and commercialization, such as grants, clinical design support, FDA fee waivers, tax incentives, and seven years of market exclusivity.