Krystal Biotech proudly announces the successful transaction of its Rare Pediatric Disease Priority Review Voucher (PRV), yielding an impressive $100 million.
This remarkable achievement stems from the commendable acceleration approval of Vyjuvek (beremagene geperpavec-svdt) by the US Food and Drug Administration (FDA). Vyjuvek has been specifically greenlit for the treatment of recessive or dominant dystrophic epidermolysis bullosa (DEB) for patients aged 6 months and beyond.
At the core of this accomplishment lies the Rare Pediatric Disease Priority Review Voucher Program, an initiative championed by the FDA. This program recognizes sponsors of rare pediatric disease product applications that meet certain stringent criteria. Its overarching objective is to incentivize the creation of novel drugs and biologics targeted at preventing and treating rare pediatric diseases. Under the program’s provisions, a PRV serves as a valuable token that can be utilized for the prioritized review of subsequent marketing applications for distinct products. Alternatively, it holds the potential for being sold or transferred, effectively becoming a strategic asset in the pharmaceutical landscape.
The divestment of the PRV for a substantial $100 million not only underscores Krystal Biotech’s dedication to innovative medical solutions but also highlights the recognition and value that its pioneering endeavors command within the industry.
This noteworthy accomplishment is a testament to the transformative power of breakthroughs in genetic medicine and the collaboration between visionary biotechnology firms and regulatory bodies. Krystal Biotech’s foray into rare pediatric diseases stands as a beacon of hope for patients and families navigating uncharted medical territories.
As Krystal Biotech propels forward, this strategic move not only adds momentum to its mission but also contributes to the advancement of therapeutic options for young patients with rare diseases. The company’s dedication to innovation and meaningful impact is now further fortified by the strategic utilization of the Rare Pediatric Disease Priority Review Voucher, a powerful tool in the journey to unlock breakthrough treatments.
