300 Billion Won? ... PRV Prices Soar, but K-Bio Is Still on the Sidelines

BioNTech to Buy PRV for $205 Million
Recent Prices Hover Around $200 Million
No Korean Company Has Received One Yet
"PRVs Should Also Be Recognized as Assets"

The price of a U.S. Food and Drug Administration (FDA) Priority Review Voucher (PRV) has soared to around 300 billion won per voucher. In addition to extending the period of patent exclusivity for a new drug, a PRV can be sold when needed to recoup research and development costs, making it a key asset in the global pharmaceutical and biotech industries. However, no Korean company has yet received a PRV, and the high barriers to development mean there is still a long way to go before the program can be used, observers say.

According to the global biotech industry on November 13, Germany's BioNTech announced that it had signed an agreement to acquire a PRV held by U.S.-based Scalarock for $205 million (about 300 billion won). A PRV is granted for a drug designated to treat conditions such as rare pediatric diseases after it receives U.S. approval. It can be used to shorten the FDA review period for another new drug from the standard 10 months to six months. BioNTech did not disclose how it would use the voucher, but industry observers believe it is most likely to be used for pumitamig, a PD-L1/VEGF bispecific antibody being co-developed with Bristol Myers Squibb (BMS).

PRV transaction prices, which had hovered around $100 million for years, have risen sharply recently. This year, PRVs have changed hands for around $200 million, including transactions involving Jazz Pharmaceuticals ($200 million), Cyprium ($205 million) and Ultragenyx ($210 million). Demand from global big pharma companies seeking to bring launches forward by about four months and extend their period of exclusive sales has driven prices higher. Launching ahead of competing drugs can also yield additional benefits, such as securing an early position in treatment guidelines and combination therapies. For major new drugs targeting annual sales of trillions of won, paying a hefty sum of around $200 million to accelerate the review process can be worthwhile.

Although the PRV has become an established asset, no Korean company has yet used one. To receive a PRV, a drug must be approved in the United States as a treatment for a rare pediatric disease or a similar condition. It must contain a new active ingredient receiving its first U.S. approval, rather than be a biosimilar or an improved drug, and developers must also obtain clinical data on patients with rare pediatric diseases, who are particularly difficult to recruit. These requirements make development challenging. Clinical trials for rare diseases are difficult to conduct using only Korean patients, making multinational trials essential. Another problem is that Korean companies often lack the financial resources to sustain long-term trials. As a result, even when they begin development, they often license their drugs to overseas partners midway through clinical trials, typically transferring the voucher to the partner as well.

In Korea, Hanmi Pharmaceutical's treatment for congenital hyperinsulinism, efpegerglucagon, and GC Biopharma and Nobel Pharma's treatment for Sanfilippo syndrome type A, GC1130A, have been designated by the FDA as drugs for rare pediatric diseases. However, there is still a long way to go before either receives an actual voucher. Efpegerglucagon is currently nearing the end of a global Phase 2 trial, while GC1130A is in a Phase 1 trial.

Experts say the Korean industry needs to change its approach, for example by specifying in contracts that it will retain ownership of the PRV even when licensing a drug to an overseas pharmaceutical company midway through clinical trials. Qurient, for instance, included a clause in its 2023 technology export agreement with the international nonprofit TB Alliance for its tuberculosis treatment telacebec, stipulating that it would retain ownership of the PRV issued upon FDA approval.

"A PRV is granted in connection with the development of a rare-disease drug, can be used in the approval process for a wide range of future new drugs, and can be freely sold. It is therefore treated as an asset in the global market," said Hwang Juri, head of external cooperation at the Korea Biotechnology Industry Organization (KoreaBIO). "Compared with this, awareness of PRVs in Korea is very low. The domestic pharmaceutical industry needs to change its culture and treat PRVs as assets, securing the rights to them from the technology licensing stage onward."

This content was produced with the assistance of AI translation services.