Annji Pharmaceutical's licensing agreement up to $250 million with US-based Avenue Therapeutics to develop and commercialise AJ201 for a rare disease has become the talk of the town, which fueled more discussions amongst Taiwanese pharmaceutical companies that have orphan drug approvals.
Rare diseases are defined in the US as diseases that affect less than 200,000 people, or diseases with a prevalence rate of 0.65 to 1 per 1,000. There are 7,000 known rare diseases affecting 350 million people worldwide, but only 200 of them have approved drug treatments.
Given the gap in the rare disease market, both the United States and the European Union have offered preferential policies to accelerate the launch of orphan drugs, such as enjoying 50% tax deduction of trial costs, exemption from processing fees, and seven to ten years of exclusive rights after the launch of new drugs. In addition to that, the lack of competitive products in the market, the high probability of success in development, high drug prices, and a gross profit margin of over 80%, multinational companies have been attracted to rush in in recent years through mergers and acquisitions or licensing.
According to an investment expert, Taiwan's pharmaceutical industry is competitive, with capital, investment and listing ratios higher than those of in Korea, Japan and India, and that new drugs and orphan drugs currently in phase II are of great interest.
Preliminary statistics show that 12 companies in Taiwan have been granted orphan drugs designation, including PharmaEssentia, PharmaEngine, TaiMed Biologics, Foresee Pharmaceuticals, Handa Pharmaceuticals, OBI Pharma, Lin BioScience, BRIM Biotechnology, Lukas Biomedical, Senhwa Biosciences, Caliway Biopharmaceuticals and Meridigen Biotechnology.
Among them, PharmaEssentia's new drugs for the treatment of polycythemia vera and PharmaEngine's new drug for pancreatic cancer have been authorised and have been successfully marketed worldwide.
TaiMed's new drug Trogarzo for the treatment of late-stage AIDS has been licensed for both the intravenous (IV) dose and the intravenous (IM) dose, while the most talked about intramuscular (IM) dose is likely to apply for sBLA marketing approval in the first half of 2023.
Handa has already obtained two orphan drug certifications for chronic myeloid leukaemia and acute lymphoblastic leukaemia, with a seven-year exclusivity.
Lin Bioscience's new drug for Streptococcus has already obtained ODD approval in the US and EU respectively, and Rare Pediatric Disease (RPD) approval in the US, and is currently approved for Phase III clinical trials in the UK and Hong Kong.
