On May 5, 2020 Mateon Therapeutics Inc. (OTCQB:MATN) reported that the US Food and Drug Administration (FDA) granted Rare Pediatric Disease Designation for CA4P/ Fosbretabulin for the treatment of of stage IIB–IV melanoma due to genetic mutations that disproportionately affect pediatric patients as a drug for a "rare pediatric disease" (Press release, Mateon Therapeutics, MAY 5, 2020, View Source [SID1234557482]).

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"We are excited about this Rare Pediatric Disease designation for our lead anti-melanoma tumor drug candidate for pediatric of stage IIB–IV melanoma patients who are in urgent need for therapeutic innovations", said Dr. Vuong Trieu, Chief Executive Officer of Mateon. "This builds on our previous Rare Pediatric Disease designation for our lead drug candidate for pancreatic cancer and glioblastoma, OT-101, for pediatric brain cancer patients (DIPG). We are looking to continuing to refocus the company efforts in the US to rare pediatric indications, while expanding our footprint to China."

Preclinical studies in which CA4P was combined with an anti-CTLA4 antibody using an EMT-6 mammary tumor model showed that 7 out of 8 mice receiving a combination of CA4P and an anti-CTLA4 antibody experienced complete remission of their tumors, compared to only 1 of 8 in the CA4P monotherapy arm and 2 of 8 in the anti-CTLA4 antibody monotherapy.

CA4P as single agent resulted in 2 Stable Disease (SD) of 5 melanoma patients who failed standard therapies. CA4P combination with carboplatin and paclitaxel resulted in one Partial Response (PR) of 6 melanoma patients who failed first-line therapy with dacarbazine and sorafenib.

Building on the single agent activity of CA4P, we are expecting that combination of CA4P with Ipilimumab or other immune-oncology drug would result in improved tumor control for the target pediatric population above the 2 PR out of 17 patients treated with Ipilimumab alone which supported the approval of Ipilimumab in pediatric melanoma.

The FDA grants rare pediatric disease designation for diseases with serious or life-threatening manifestations that primarily affect people aged from birth to 18 years, and that affect fewer than 200,000 people in the U.S. Under the FDA’s Rare Pediatric Disease Priority Review Voucher program, a sponsor who receives an approval of a new drug application or biologics license application for a product for the prevention or treatment of a rare pediatric disease may be eligible for a voucher, which can be redeemed to obtain priority review for any subsequent marketing application, and may be sold or transferred. In August 2019, AstraZeneca has reportedly paid approximately $95 million to buy a priority review voucher from Swedish Orphan Biovitrum (Sobi) (View Source). Likewise, Biohaven Pharmaceutical Holding Company Ltd. reportedly paid approximately $105 million for a priority review voucher in March 2019 (View Source).