Jazz Pharmaceuticals Plc

iStock/ A-Tom A renewal of the FDA’s pediatric rare disease voucher program is couched within a massive $1.2 trillion spending bill the House is set to vote on later this week. There is hope yet for the FDA’s rare pediatric disease priority vouchers as key legislation is set to come before the U.S. House of Representatives later this week and the Senate next week. If passed, it would renew the program and make these expedited review tickets available to drugmakers once again. The House Appropriations Committee on Tuesday released the text of a funding package to keep the government open after a Jan. 30 deadline. That package includes $1.2 trillion in spending across the federal government, including Defense, Transportation, Housing and Urban Development and Health and Human Services. The rare pediatric priority review voucher (PRV) program —now up for renewal in the HHS section of the legislation—first came into play in 2012 to encourage drugmakers to develop therapies for rare pediatric diseases. As a reward for developing and securing an approval for such conditions, companies are given a voucher that they can claim to speed up the review of a different drug application. Companies can also choose to sell their vouchers for some liquidity. Last week, for instance, Jazz Pharmaceuticals offloaded a ticket for $200 million , the highest sale price in nearly 10 years. In September 2024, Congress failed to authorize the renewal of the program, forcing the FDA in December of that year to wind down the scheme. Review vouchers have become more expensive as the program expired. Then, in September last year, the House Committee on Energy and Commerce reignited hopes for the program after it advanced a bill that would reauthorize the awards. The proposed legislation was dubbed the Mikaela Naylon Give Kids a Chance Act of 2025 and, if passed, would keep the vouchers going through Sept. 30, 2029. A vote on the spending proposal is expected to take place later this week, according to reporting from The Washington Post . Also of interest to the pharma sector is the inclusion in the spending bill of a series of reforms to pharmacy benefits managers (PBMs), including a new HHS review of fees paid to PBMs and a requirement that PBMs submit reports to HHS covering drug pricing and rebate data, as well as written justifications for coverage choices. The overall funding package is designed to prevent another government shutdown and would keep federal offices running past the Jan. 31 deadline, as per the Post . The proposal also puts the National Institutes of Health’s budget at $48.7 billion—a $415 million increase from 2025 and in direct contrast with the Trump administration’s thrust to cut the agency’s appropriation by $18 billion . If the spending bill passes the House vote, it will go on to the Senate next, though no schedule for this deliberation has yet been announced.

The House is set to vote this week on a bipartisan bill to keep the government open beyond Jan. 30, with new proposals to change how pharmacy benefit managers operate and to reauthorize a lapsed FDA voucher program for rare pediatric drugs. The spending bill, which the Senate is expected to vote on next week, features PBM reforms that have been floated for years, including requirements that HHS review the fees or other forms of compensation paid to PBMs or their affiliates. The bill would also require PBMs to give HHS and insurers a new annual report that includes drug pricing and rebate data, as well as written justifications in several areas — such as for providing more favorable coverage for a reference biologic rather than a biosimilar. The Centers for Medicare & Medicaid Services would receive an additional $188 million from the bill to carry out the PBM reforms. The spending bill also would reauthorize the rare pediatric priority review voucher (PRV) program, which stalled in the Senate last December and has lingered since 2024. The program would be extended through Sept. 30, 2029, if the House version of the spending bill remains as is. The transferable vouchers were created in 2012 to incentivize new drugs to treat rare pediatric illnesses, including rare cancers and genetic diseases. The vouchers can be used to speed approvals of future drugs and can be sold. Last week, Jazz Pharmaceuticals sold a rare pediatric PRV for $200 million. The bill also includes provisions related to the requirement that companies run pediatric studies after winning approval for drugs in adult populations. New language in the bill seeks to ensure the FDA wouldn’t take enforcement action against drugs or biologics that are no longer marketed, among other provisions. And contrary to President Donald Trump’s budget that sought to slash NIH funding by $18 billion, or 40%, the NIH under the House bill would receive $400 million more than it received in 2025. That increase includes $128 million more than 2025 for cancer research and a $100 million increase for research into Alzheimer’s and associated dementias.

近日，Jazz Pharmaceuticals公司宣布，已将其获得的优先审评券（PRV）“以2亿美元的价格”卖给了一位未透露姓名的买家，这是目前已知近十年来“优先审凭券”卖出的最高成交价。 该张优先审评券为其罕见脑瘤药物Modeyso去年8月获得FDA批准上市之后获得。 Modeyso是一种口服蛋白酶激活剂，被FDA批准用于治疗携带H3 K27M突变弥漫性中线神经胶质瘤。去年3月，Jazz通过收购Chimerix公司（9.35亿美元）获得了Modeyso。 该公司透露，其将获得该笔交易一半的净收入。 市场行情的波动 优先审评券是旨在通过提供可兑换或出售以获得不同产品优先审评的券，激励罕见儿科疾病新药、以及热带疾病的开发。 在新药研发早期（Pre-clinical阶段），企业可向FDA申请优先审评资格认定，在该新药研发成功被FDA批准上市后，可凭优先审评资格认定向FDA兑换优先审评券。这种审评券可以无限制转让和销售，可用于加速其他非罕见病药物的审评过程(10个月缩短至约6个月)。 截止目前，优先审评券的最高成交价约为3.5亿美元。2015年，AbbVie以3.5亿美元的价格从United Therapeutics公司手中买下了这张高价优先审评券。 然而，随着FDA发放的优先审评券数量的增加，其经历了行情的下跌，成交价也随之下滑。 收紧优先审评券计划 另一方面，美国国会有意在收紧优先审评券计划。 FDA发放的优先审评券数量的日益增多，也引起了一些参议员的严重不满。Bernie Sanders在内的参议员认为该计划可能被滥用，未能有效推动真正有价值的儿科罕见病药物的研发。 在2024年9月，美国国会便未续期“罕见儿科疾病优先审评券计划”（该计划需定期重新获得授权），这使得FDA不得不停止继续发放优先审评券。 在该计划未被得到重新授权后，依据现行法规，除了在2024年12月20日前已经获得罕见儿科疾病认定的药物之外，否则FDA不能发放任何新的优先审评券。而且拥有罕见儿科疾病认定的公司只有在2026年9月30日之前取得上市批准，才能兑换成优先审评券。 而到2025年，该计划的续期仍遭到了参议院的阻拦。 尽管在2025年9月的时候，美国众议院通过了对“续期罕见儿科疾病优先审评券计划”的立法提案，但由于参议员之间出现分歧，导致未成功将该提案推进相关立法，使得最终该计划又未能如期续期。 这使得FDA发放的优先审查券数量大幅减少，市场行情则因此出现了显著上涨，去年，优先审评券的成交价平均在1.5亿美元左右。 随着该计划续期再一次推迟，未来市面上可以买卖的优先审查券将进一步减少，交易价也可能保持持续上涨的趋势。 参考出： https://www.biospace.com/business/jazz-sells-priority-review-voucher-for-200m-as-programs-renewal-languishes-in-senate https://investor.jazzpharma.com/static-files/650e516c-58b5-4e36-8785-b1cf05e0c25e#:~:text=%E2%80%A2%20Future%20cash%20taxes%20reduced%20by%20~%24200M,sell%20PRV%20for%20%24200M%20in%20gross%20proceeds2