Pharma Focus Asia

Abbisko Therapeutics Has Been Granted Orphan Drug Designation by the European Medicines Agency (EMA) for Its CSF-1R Inhibitor Pimicotinib (ABSK021)

Tuesday, January 09, 2024

Abbisko Therapeutics (HKEX code: 02256) has recently announced that its experimental CSF-1R inhibitor, pimicotinib (ABSK021), has been granted orphan drug designation (ODD) by the European Medicines Agency (EMA) for the treatment of inoperable tenosynovial giant cell tumor (TGCT).

This ODD approval from the EMA comes with various incentives, such as protocol assistance, fee reductions, procedural advantages for market authorization, and market exclusivity. Member states in the European Union may also offer specific incentives for orphan drugs.

In June 2023, pimicotinib received the Priority Medicines (PRIME) designation from the EMA, aiming to expedite the review process for promising medicines addressing unmet medical needs.

In December 2023, Abbisko entered into an exclusive licensing agreement with Merck for the commercialization of pimicotinib in the Chinese mainland, Hong Kong, Macau, and Taiwan. Merck also obtained an exclusive option for global commercial rights, subject to agreed terms and conditions. The licensing agreement involves a one-time, non-refundable down payment of US$ 70 million to Abbisko. If Merck exercises the global commercialization option, Abbisko will receive an additional option exercising fee. The total aggregate payments, including upfront, option exercising, and development/commercialization milestones, will amount to US$ 605.5 million. Abbisko will also be entitled to double-digit percentage royalties on annual net sales.

Pimicotinib, an orally available CSF-1R inhibitor developed independently by Abbisko Therapeutics, has received Breakthrough Designation (BTD) and PRIME designation from China NMPA, U.S. FDA, and EMA for treating TGCT patients ineligible for surgery. This marks the first simultaneous global Phase III clinical trial for TGCT conducted in China, the U.S., Canada, and Europe.

Results from a Phase 1b trial for TGCT, with a 1-year follow-up, showcased pimicotinib's impressive 87.5% Overall Response Rate (ORR) in the 50 mg QD cohort (28/32, including 3 complete responses), presented at the 2023 CTOS. Pimicotinib has successfully completed a Phase I dose-escalation trial in the U.S. and was granted Fast Track Designation (FTD) by the U.S. FDA in December of the same year for the treatment of unresectable TGCT.

Beyond TGCT, Abbisko is actively investigating pimicotinib's potential in treating various solid tumors, with Phase II clinical studies approved by China NMPA for chronic graft-versus-host disease (cGVHD) and advanced pancreatic cancer. As of now, no highly selective CSF-1R inhibitors have received approval in China.



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