Novartis Acquires FDA Priority Review Voucher from bluebird bio for $103 Million

According to FIERCEPharma, Novartis has reached an agreement with bluebird bio to acquire a U.S. FDA Priority Review Voucher (PRV) for $103 million. However, the completion of this $103 million transfer agreement depends on the FDA's approval of bluebird bio's gene therapy, LentiGlobin, for the treatment of sickle cell disease (SCD). The FDA is expected to make a decision on bluebird bio's LentiGlobin application by December 20th.

The Priority Review Voucher program is a benefit system implemented by the U.S. FDA to incentivize pharmaceutical companies to develop drugs targeting certain tropical diseases, rare diseases, and medical countermeasures for substance threats. Pharmaceutical companies that successfully develop drugs for diseases on the specified list are eligible to receive a PRV, which can be used by the company itself or sold to other pharmaceutical companies. The voucher allows the recipient to expedite the review of a new drug application, reducing the standard 10-month review time to 6 months.

Following argenx and BMS, Novartis becomes the third buyer to participate in this PRV transfer.

According to a document from bluebird bio, Novartis is another company involved in the PRV transfer agreement. If the transaction is completed, it will be bluebird bio's third PRV sale in the past year.

Bluebird bio obtained two PRVs in 2022 for the FDA approval of ZYNTEGLO (betibeglogene autotemcel) for the treatment of adult and pediatric patients with transfusion-dependent beta-thalassemia, and SKYSONA (elivaldogene autotemcel) for the treatment of early-stage active cerebral adrenoleukodystrophy (CALD).

Subsequently, in November 2022 and January 2023, bluebird bio successfully monetized the PRVs by selling them to argenx, a Dutch immunology company, and multinational giant BMS, for $102 million and $95 million, respectively. The PRV purchased by argenx is expected to be used for the future marketing application of their first-in-class neonatal Fc receptor (FcRn) antagonist, efgartigimod.

Insufficient funding for less than a year

Transfer of PRV expected to generate cash flow

Bluebird bio revealed in its second-quarter earnings report that the company is currently facing financial challenges. As of June 30, 2023, including restricted funds, bluebird's cash position was $291 million, and the company expects cash consumption to be between $270 million and $300 million for the full year. Without the restricted cash of approximately $45 million, the company's cash runway would only be sustained until the second quarter of 2024.

Chris Krawtschuk, CFO of bluebird bio, stated in a statement that the sale of the LentiGlobin PRV would provide "an important non-dilutive source of capital for bluebird bio ahead of the anticipated launch."

Meanwhile, with the launch of Zynteglo and Skysona, bluebird bio expects its cash flow to continue until mid-2024. However, the road to market for these two products has been challenging.

Zynteglo, bluebird bio's first proprietary product in gene therapy after 27 years of research and development, became the first FDA-approved ex vivo lentiviral gene therapy in August 2022. It is expected to change the treatment paradigm for beta-thalassemia, which currently requires lifelong care. Adult and pediatric patients with the disease require regular red blood cell transfusions.

Due to regulatory oversight of drug specifications and manufacturing processes in Europe, the commercialization process of Zynteglo, which was born in 2019, was delayed multiple times until it was postponed to 2020. It coincided with the global outbreak of the COVID-19 pandemic, further pushing back the first commercial treatment to the second half of the year.

Fortunately, in September 2022, bluebird bio received good news. The gene therapy Skysona, after being approved by the European Union, was granted accelerated approval by the U.S. FDA for the treatment of boys aged 4 to 17 with early-stage cerebral adrenoleukodystrophy (CALD). Skysona utilizes a lentiviral vector to introduce functional copies of the ABCD1 gene into the patient's own hematopoietic stem cells ex vivo, which are then reinfused into the patient to express the ALD protein, thereby preventing the progression of CALD and preserving neurological function as much as possible. The therapy is priced at $3 million.

Based on the above information, an original article with a similarity rate of less than 50% is provided below:

Novartis has acquired a Priority Review Voucher (PRV) from bluebird bio for $103 million, pending FDA approval of bluebird bio's gene therapy, LentiGlobin, for the treatment of sickle cell disease (SCD). The FDA is expected to make a decision on LentiGlobin by December 20th. The PRV program is a FDA incentive program that allows pharmaceutical companies to expedite the review of a new drug application. Novartis is the third company to participate in a PRV transfer, following argenx and BMS. Bluebird bio obtained two PRVs in 2022 and sold them to argenx and BMS for $102 million and $95 million, respectively. The sale of the PRV will provide bluebird bio with much-needed capital. The company is facing financial challenges and expects its cash runway to last until the second quarter of 2024. The launch of Zynteglo and Skysona, bluebird bio's gene therapies for beta-thalassemia and CALD, is expected to generate cash flow, but the commercialization process has been delayed. Zynteglo was approved by the FDA in August 2022 and Skysona received accelerated approval in September 2022.