The US Food and Drug Administration (FDA) has received an orphan drug designation (ODD) for the leading product, berubicin (RTA 744; CNS Pharmaceuticals, Houston, TX).
Under a prior developer, berubicin was granted ODD by the FDA for the treatment of malignant gliomas. In the prior developer's phase 1 trial of berubicin (NCT00538343), 44% of the participants demonstrated a significant improvement in progression free survival. Additionally, 1 participant in this study experienced a complete response to his treatment with berubicin.
"We are excited to announce the ODD application submission for berubicin, as it would grant special status and accelerate the development of berubicin to treat glioblastoma, one of the world's most aggressive forms of cancer," stated John Climaco, chief executive officer of CNS Pharmaceuticals. "We are pleased to continue to execute upon our strategic initiatives and submit our application within the anticipated timeline outlined within our previous filings. We feel cautiously optimistic about the application, given the past ODD of the molecule and positive phase 1 results. We look forward to initiating a phase 2 trial evaluating the effect of berubicin on patients with glioblastoma later this year."
The Orphan Drug Act (ODA) allows granting special status to a drug or biological product for a rare disease or condition upon request of a sponsor. This status is referred to as orphan designation sometimes referred to as orphan status.
The FDA grants ODD status to products that treat rare diseases, providing incentives to sponsors developing drugs or biologics. The FDA defines rare diseases as those affecting fewer than 200,000 individuals in the US at any given time. Because of small patient numbers, treatment for these rare diseases would not be considered economically feasible without government programs to support their economic viability. The ODD would qualify berubicin for certain benefits and incentives, including 7 years of marketing exclusivity if regulatory approval is ultimately received for the designated indication, potential tax credits for certain activities, eligibility for orphan drug grants, and the waiver of certain administrative fees. The receipt of ODD status does not change the regulatory requirements or process for obtaining marketing approval.
James Geyer, MD, and Paul Cox
Natalia P. Rocha, PharmD, MSc, PhD; Gabriela D. Colpo, PhD; Antonio L. Teixeira, MD, PhD, MSc; and Erin Furr Stimming, MD
Claire Smyth, BSc; David Roberts, BSc; and Kenneth Monaghan, PhD