Latest news with #RarePediatricDiseasePriorityReviewVoucher
Yahoo
12-05-2025
- Business
- Yahoo
Abeona Therapeutics® Enters into Agreement to Sell Priority Review Voucher for $155 Million
CLEVELAND, May 12, 2025 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (Nasdaq: ABEO) today announced it has entered into a definitive asset purchase agreement to sell its Rare Pediatric Disease Priority Review Voucher (PRV) for gross proceeds of $155 million upon the closing of the transaction. Abeona was awarded the PRV following the U.S. Food and Drug Administration (FDA) approval of ZEVASKYN™ (prademagene zamikeracel) on April 28, 2025. 'With proceeds from this PRV sale, we have sufficient cash for more than two years of operating expenses without the need for capital infusion and not accounting for ZEVASKYN sales,' said Joe Vazzano, Chief Financial Officer of Abeona. 'Furthermore, with ZEVASKYN becoming available to treat patients beginning third quarter of 2025, we anticipate becoming profitable in early 2026.' The transaction is subject to customary closing conditions, including expiration of the applicable waiting period under the Hart-Scott Rodino (HSR) Antitrust Improvements Act. Stifel was lead financial advisor to Abeona on the transaction. Jefferies also served as financial advisor on the transaction. About Abeona Therapeutics Abeona Therapeutics Inc. is a commercial-stage biopharmaceutical company developing cell and gene therapies for serious diseases. Abeona's ZEVASKYN™ (prademagene zamikeracel) is the first and only autologous cell-based gene therapy for the treatment of wounds in adults and pediatric patients with recessive dystrophic epidermolysis bullosa (RDEB). The Company's fully integrated cell and gene therapy cGMP manufacturing facility in Cleveland, Ohio serves as the manufacturing site for ZEVASKYN commercial production. The Company's development portfolio features adeno-associated virus (AAV)-based gene therapies for ophthalmic diseases with high unmet medical need. Abeona's novel, next-generation AAV capsids are being evaluated to improve tropism profiles for a variety of devastating diseases. For more information, visit ZEVASKYNTM, Abeona AssistTM, Abeona Therapeutics®, and their related logos are trademarks of Abeona Therapeutics Inc. Forward-Looking Statements This press release contains certain statements that are forward-looking within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended, and that involve risks and uncertainties. We have attempted to identify forward-looking statements by such terminology as 'may,' 'will,' 'believe,' 'anticipate,' 'expect,' 'intend,' 'potential,' and similar words and expressions (as well as other words or expressions referencing future events, conditions or circumstances), which constitute and are intended to identify forward-looking statements. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, numerous risks and uncertainties, including but not limited to, our ability to successfully generate commercial sales of ZEVASKYN and generate future revenue; the successful closing of our sale transaction for the Priority Review Voucher; continued interest in our rare disease portfolio; our ability to enroll patients in clinical trials; the outcome of future meetings with the FDA or other regulatory agencies, including those relating to preclinical programs; the ability to achieve or obtain necessary regulatory approvals; the impact of any changes in the financial markets and global economic conditions; risks associated with data analysis and reporting; and other risks disclosed in the Company's most recent Annual Report on Form 10-K and subsequent periodic reports filed with the Securities and Exchange Commission. The Company undertakes no obligation to revise the forward-looking statements or to update them to reflect events or circumstances occurring after the date of this press release, whether as a result of new information, future developments or otherwise, except as required by the federal securities laws. CONTACT: Investor and Media Contact: Greg Gin VP, Investor Relations and Corporate Communications Abeona Therapeutics ir@ in to access your portfolio
Time of India
28-04-2025
- Business
- Time of India
Roche made over 100 times the cost of a medicine that it sold in India, calculations show; company defends itself
Pharmaceutical giant Roche may have made profits more than a hundred times what it cost the company to bring a rare-disease drug to the Indian market, The Times of India reported on April 28. The drug in question is risdiplam , a medication for spinal muscular atrophy (SMA). The report on Roche's profit estimates are based on evaluations by public health advocates. #Pahalgam Terrorist Attack India stares at a 'water bomb' threat as it freezes Indus Treaty India readies short, mid & long-term Indus River plans Shehbaz Sharif calls India's stand "worn-out narrative" Following Roche's refusal to disclose details about the expenses incurred during clinical trials, Knowledge Ecology International (KEI), a non-profit organisation based in the United States that focuses on making medicines more accessible, performed their own calculations, ToI's report (by Rema Nagarajan) said. They estimated the total cost of the trials to be approximately $50 million. Roche's revenue from risdiplam sales between 2021 and 2024 amounted to $5.8 billion, equating to nearly 115 times the calculated trial expenses. by Taboola by Taboola Sponsored Links Sponsored Links Promoted Links Promoted Links You May Like Elegant New Scooters For Seniors In 2024: The Prices May Surprise You Mobility Scooter | Search Ads Learn More Undo In reply to the figures provided by KEI, Roche explained, "Only a small fraction of projects succeed in going 'from bench to bedside,' and we must also account for the costs associated with unsuccessful projects, whereas your calculation only considers successful developments. Our commitment to innovation is also reflected in our ongoing efforts to develop next-generation therapies, as evidenced by our continuous work on new treatments for SMA." The three risdiplam trials referenced by the US Food and Drug Administration (FDA), along with four previous clinical trials, involved a total of 587 participants. KEI arrived at the estimated cost per patient for the trials by synthesizing data from multiple sources regarding the expenses associated with clinical trials. In the United States, there is a tax incentive for qualifying clinical trials related to rare diseases. Live Events When KEI applied this tax credit to the trial expenses, the net costs were estimated to range from $11 million to $25 million. Typically, clinical trials account for about 60-70% of the total costs involved in drug development. Additionally, Roche received a Rare Pediatric Disease Priority Review Voucher (PRV) in August 2020. This voucher serves as an incentive aimed at promoting the development of drugs and biologics for serious or life-threatening illnesses and can be sold to third parties. In 2020, the estimated market value of a PRV was around $100 million, which exceeds Roche's spending on clinical trials, as noted by KEI. Since its inception in 2003, the Spinal Muscular Atrophy Foundation (SMAF) has invested $100 million into the development of treatments for SMA. Roche acquired an exclusive global license for PTC Therapeutics' SMA program for $30 million, which was developed in collaboration with SMAF. Roche's manufacturing costs for risdiplam are relatively low, as a year's worth of treatment requires less than 2 grams of the drug. KEI estimates that Roche could produce risdiplam for under $50,000 per kilo, or $50 per gram. In India, the price of risdiplam treatment amounts to approximately Rs 72 lakh per adult patient annually, a cost that is beyond the means of many patients. According to Roche's statement to the Times of India, as of October 2024, out of the 168 patients receiving SMA treatment from Roche, 56 were being treated at no cost through the company's Compassionate Use Program (CUP). Additionally, 53 patients were covered under various government schemes, including the Central Government Health Scheme, Defence, Employee State Insurance (ESI), and Railways. The remaining 59 patients accessed the medication through Roche's Patient Access Program, initiated in 2021, which allows Roche to provide one free bottle of treatment for every purchased bottle. Cure SMA, an organisation established by parents of children diagnosed with SMA, has 1,800 registered patients, while the government portal for rare diseases lists 700 registered SMA patients.
Yahoo
07-04-2025
- Business
- Yahoo
Zevra Announces Closing of Sale of Rare Pediatric Disease Priority Review Voucher for $150 Million
CELEBRATION, Fla., April 07, 2025 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra, or the Company), a commercial-stage company focused on providing therapies for people living with rare disease, today announced the closing of the sale of its Rare Pediatric Disease Priority Review Voucher (PRV) for gross proceeds of $150 million. 'We have reached another key milestone with the closing on the sale of our PRV, which we received following the successful approval of MIPLYFFATM (arimoclomol) from the U.S. Food and Drug Administration (FDA),' said LaDuane Clifton, Zevra's Chief Financial Officer. 'The $150 million in gross proceeds is non-dilutive capital that further enhances our financial flexibility to support our strategic priorities, which includes executing on the commercial launches of MIPLYFFA and OLPRUVA®, supporting our ongoing Phase 3 trial for celiprolol, and augmenting our solid balance sheet to support future investments that are in alignment with our strategic plan.' Zevra was granted the PRV in September 2024 by the FDA, in connection with the FDA's approval of MIPLYFFA, the first U.S. approved treatment for the ultra-rare neurodegenerative disease, Niemann-Pick type C. In addition, the Company reported that available unaudited cash, cash equivalents and investments as of Mar. 31, 2025, were $68.7 million. Combined with the cash proceeds of $148.3 million, net of fees, from the sale of the PRV after the end of the quarter, cash, cash equivalents and investments would be $217.0 million. About Zevra Therapeutics, Inc. Zevra Therapeutics, Inc. is a commercial-stage company combining science, data, and patient need to create transformational therapies for rare diseases with limited or no treatment options. Our mission is to bring life-changing therapeutics to people living with rare diseases. With unique, data-driven development and commercialization strategies, the Company is overcoming complex drug development challenges to make new therapies available to the rare disease community. For more information, please visit or follow us on X and LinkedIn. Financial Disclosure Advisory The cash, cash equivalents and investments information provided in this press release is based on preliminary unaudited information and management estimates for the quarter ended March 31, 2025, is not a comprehensive statement of the Company's financial results as of and for the fiscal quarter ended March 31, 2025 or any other period, and is subject to completion of the Company's financial closing procedures. The Company's independent registered public accounting firm has not conducted a review of and does not express an opinion or any other form of assurance with respect to this preliminary estimate. Cautionary Note Concerning Forward-Looking Statements This press release may contain forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking statements include all statements that do not relate solely to historical or current facts, including without limitation statements regarding the potential benefits of any of our products or product candidates for any specific disease or at any dosage; the progress of our clinical trials, our strategic and product development objectives; the benefits of the transaction and its impact on the Company's balance sheet or strategic goals; our financial position, including our cash, cash equivalents and investments and available resources; and the timing of any of the foregoing. Forward-looking statements are based on information currently available to Zevra and its current plans or expectations. They are subject to several known and unknown uncertainties, risks, assumptions, and other important factors that may cause our actual results, performance, or achievements to be materially different from any future results, performance, or achievements expressed or implied by the forward-looking statements. Given these risks and uncertainties, you should not place undue reliance on these forward-looking statements. These and other important factors are described in detail in the "Risk Factors" section of Zevra's Annual Report on Form 10-K for the year ended December 31, 2024, and Zevra's other filings with the Securities and Exchange Commission. While we may elect to update such forward-looking statements at some point in the future, except as required by law, we disclaim any obligation to do so, even if subsequent events cause our views to change. Although we believe the expectations reflected in such forward-looking statements are reasonable, we cannot assure that such expectations will prove correct. These forward-looking statements should not be relied upon as representing our views as of any date after the date of this press release. Zevra Contact Nichol Ochsner+1 (732) 754-2545nochsner@ Media Contact Russo PartnersDavid Schull+1 (858) in to access your portfolio
Yahoo
27-02-2025
- Business
- Yahoo
Zevra Therapeutics Enters Agreement to Sell its Rare Pediatric Disease Priority Review Voucher for $150 Million
CELEBRATION, Fla., Feb. 27, 2025 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra, or the Company), a commercial-stage company focused on providing therapies for people living with rare disease, today announced that it has entered into a definitive asset purchase agreement to sell its Rare Pediatric Disease Priority Review Voucher (PRV) for gross proceeds of $150 million upon the closing of the transaction, which is expected to take place within 30 to 45 days, subject to customary closing conditions. LaDuane Clifton, Zevra's Chief Financial Officer said, 'This non-dilutive capital strengthens our balance sheet by adding gross cash proceeds of $150 million, supporting continued investment in our strategic priorities, which include executing the commercial launches of MIPLYFFA™ and OLPRUVA®, and advancing our pipeline of product candidates to address unmet needs within the rare disease community.' The PRV was granted to Zevra in September 2024 following approval by the U.S. Food and Drug Administration of MIPLYFFA (arimoclomol), which is indicated for use in combination with miglustat for the treatment of neurological manifestations of Niemann-Pick disease type C (NPC) in adult and pediatric patients 2 years of age and older. The transaction is subject to customary closing conditions, including expiration of the applicable waiting period under the Hart-Scott Rodino Antitrust Improvements Act (HSR). Cantor Fitzgerald acted as Zevra's exclusive financial advisor and Latham & Watkins LLP acted as Zevra's legal advisor for this transaction. About Zevra Therapeutics, Inc. Zevra Therapeutics, Inc. is a commercial-stage company combining science, data, and patient need to create transformational therapies for rare diseases with limited or no treatment options. Our mission is to bring life-changing therapeutics to people living with rare diseases. With unique, data-driven development and commercialization strategies, the Company is overcoming complex drug development challenges to make new therapies available to the rare disease community. For more information, please visit or follow us on X and LinkedIn. Cautionary Note Concerning Forward-Looking Statements This press release may contain forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking statements include all statements that do not relate solely to historical or current facts, including without limitation statements regarding the potential benefits of any of our products or product candidates for any specific disease or at any dosage; our strategic and product development objectives; the consummation and benefits of the transaction and its impact on the Company's balance sheet; the outcome of any required filings under the HSR; prescription enrollments; our ability to support patients as they navigate the benefits verification process to obtain either MIPLYFFA™ or OLPRUVA®; availability of and access to MIPLYFFA and OLPRUVA; and the timing of any of the foregoing. Forward-looking statements are based on information currently available to Zevra and its current plans or expectations. They are subject to several known and unknown uncertainties, risks, assumptions, and other important factors that may cause our actual results, performance, or achievements to be materially different from any future results, performance, or achievements expressed or implied by the forward-looking statements. Given these risks and uncertainties, you should not place undue reliance on these forward-looking statements. These and other important factors are described in detail in the "Risk Factors" section of Zevra's Annual Report on Form 10-K for the year ended December 31, 2023, Zevra's Quarterly Report on Form 10-Q for the three and nine months ended September 30, 2024, and Zevra's other filings with the Securities and Exchange Commission]. While we may elect to update such forward-looking statements at some point in the future, except as required by law, we disclaim any obligation to do so, even if subsequent events cause our views to change. Although we believe the expectations reflected in such forward-looking statements are reasonable, we cannot assure that such expectations will prove correct. These forward-looking statements should not be relied upon as representing our views as of any date after the date of this press release. _____________________________________________ Zevra Contact Nichol Ochsner+1 (732) 754-2545nochsner@ Russo Partners Contact David Schull+1 (858) in to access your portfolio
