Ultragenyx Receives Breakthrough Therapy Designation for Setrusumab (UX143) in Osteogenesis Imperfecta
Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) has received Breakthrough Therapy Designation from the FDA for setrusumab (UX143) to reduce fracture risk in patients 2 years and older with osteogenesis imperfecta (OI) Type I, III, or IV. This designation is based on promising clinical evidence from the Phase 2 portion of the Orbit study and the completed Phase 2b ASTEROID study, which showed a rapid and significant decrease in fracture rates.
Dr. Eric Crombez, chief medical officer at Ultragenyx, emphasized the importance of this designation in recognizing the seriousness of OI and the potential impact of setrusumab. The Breakthrough Therapy Designation aims to expedite development and review of drugs for serious diseases that show substantial improvement over existing therapies. Setrusumab has also received Orphan Drug Designation in the US and EU, rare pediatric disease designation in the US, and acceptance into the EMA's Priority Medicines program.
Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) ha ricevuto la Designazione di Terapia Innovativa dalla FDA per setrusumab (UX143) per ridurre il rischio di fratture nei pazienti di 2 anni e oltre affetti da osteogenesi imperfetta (OI) di tipo I, III o IV. Questa designazione si basa su evidenze cliniche promettenti provenienti dalla fase 2 dello studio Orbit e dallo studio ASTEROID fase 2b completato, che ha mostrato una diminuzione rapida e significativa delle fratture.
Il Dr. Eric Crombez, direttore medico di Ultragenyx, ha sottolineato l'importanza di questa designazione per riconoscere la gravità dell'OI e il potenziale impatto di setrusumab. La Designazione di Terapia Innovativa mira a velocizzare lo sviluppo e la revisione di farmaci per malattie gravi che mostrano miglioramenti sostanziali rispetto alle terapie esistenti. Setrusumab ha inoltre ricevuto la Designazione di Farmaco Orfano negli USA e nell'UE, la designazione di malattia pediatrica rara negli USA e l'accettazione nel programma EMA Priority Medicines.
Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) ha recibido la Designación de Terapia Innovadora por parte de la FDA para setrusumab (UX143) con el fin de reducir el riesgo de fracturas en pacientes de 2 años o más con osteogénesis imperfecta (OI) tipo I, III o IV. Esta designación se basa en evidencia clínica prometedora del estudio Orbit en su fase 2 y del estudio ASTEROID fase 2b completado, que mostraron una disminución rápida y significativa en las tasas de fracturas.
El Dr. Eric Crombez, director médico de Ultragenyx, enfatizó la importancia de esta designación para reconocer la gravedad de la OI y el potencial impacto de setrusumab. La Designación de Terapia Innovadora tiene como objetivo acelerar el desarrollo y la revisión de medicamentos para enfermedades graves que muestran mejoras sustanciales sobre las terapias existentes. Setrusumab también ha recibido la Designación de Medicamento Huérfano en EE. UU. y la UE, designación de enfermedad pediátrica rara en EE. UU., así como aceptación en el programa de Medicamentos Prioritarios de la EMA.
Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE)는 FDA로부터 세트루수맙(UX143)에 대해 골형성부전증(OI) 1형, 3형 또는 4형 환자 2세 이상에서 골절 위험을 줄이기 위한 획기적 치료제 designation을 받았습니다. 이 designation은 Orbit 연구의 2단계 부분과 완료된 2b 단계 ASTEROID 연구의 유망한 임상 근거를 바탕으로 하며, 이는 골절률의 빠르고 상당한 감소를 보여주었습니다.
Ultragenyx의 최고 의학 책임자인 Eric Crombez 박사는 OI의 심각성을 인식하고 세트루수맙의 잠재적 영향을 강조하였습니다. 획기적 치료제 designation은 기존 치료법에 비해 상당한 개선을 보이는 중병 치료제의 개발 및 검토를 가속화하는 것을 목표로 합니다. 세트루수맙은 미국 및 EU에서 고아약 designation, 미국에서 드문 소아질환 designation, 및 EMA의 우선 의약품 프로그램에 수용되었습니다.
Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) a reçu la désignation de thérapie innovante de la part de la FDA pour le setrusumab (UX143) afin de réduire le risque de fractures chez les patients de 2 ans et plus atteints d'ostéogenèse imparfaite (OI) de type I, III ou IV. Cette désignation est fondée sur des preuves cliniques prometteuses provenant de la phase 2 de l'étude Orbit et de l'étude ASTEROID de phase 2b achevée, qui a montré une diminution rapide et significative des taux de fractures.
Le Dr Eric Crombez, directeur médical d'Ultragenyx, a souligné l'importance de cette désignation pour reconnaître la gravité de l'OI et l'impact potentiel du setrusumab. La désignation de thérapie innovante vise à accélérer le développement et l'examen des médicaments pour des maladies graves qui montrent une amélioration substantielle par rapport aux thérapies existantes. Le setrusumab a également reçu la désignation de médicament orphelin aux États-Unis et dans l'UE, la désignation de maladie pédiatrique rare aux États-Unis, ainsi que l'acceptation dans le programme de médicaments prioritaires de l'EMA.
Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) hat von der FDA die Bezeichnung als Durchbruchtherapie für Setrusumab (UX143) erhalten, um das Frakturrisiko bei Patienten ab 2 Jahren mit Osteogenesis imperfecta (OI) Typ I, III oder IV zu reduzieren. Diese Bezeichnung basiert auf vielversprechenden klinischen Beweisen aus dem Phase-2-Teil der Orbit-Studie sowie der abgeschlossenen Phase-2b ASTEROID-Studie, die einen schnellen und signifikanten Rückgang der Frakturraten zeigte.
Dr. Eric Crombez, Chief Medical Officer von Ultragenyx, betonte die Bedeutung dieser Bezeichnung zur Anerkennung der Schwere der OI und des potenziellen Einflusses von Setrusumab. Die Bezeichnung als Durchbruchtherapie zielt darauf ab, die Entwicklung und Überprüfung von Arzneimitteln für schwere Krankheiten zu beschleunigen, die erhebliche Verbesserungen gegenüber bestehenden Therapien zeigen. Setrusumab hat auch die Orphan Drug Designation in den USA und der EU, die Bezeichnung als seltene pädiatrische Erkrankung in den USA und die Aufnahme in das EMA-Programm für prioritäre Arzneimittel erhalten.
- Received FDA Breakthrough Therapy Designation for setrusumab (UX143)
- Positive clinical evidence from Phase 2 Orbit study showing rapid decrease in fracture rates
- Potential expedited development and review process for setrusumab
- Multiple designations received (Orphan Drug, rare pediatric disease, PRIME) supporting the drug's importance
- None.
NOVATO, Calif., Oct. 07, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, today announced that it has received Breakthrough Therapy Designation from the U.S. Food and Drug Administration (FDA) for setrusumab (UX143) as a treatment to reduce the risk of fracture associated with osteogenesis imperfecta (OI) Type I, III, or IV in patients 2 years of age and older.
"FDA designation of setrusumab as a Breakthrough Therapy emphasizes the seriousness of osteogenesis imperfecta and the impact of this disease on people and their families affected by this disorder,” said Eric Crombez, M.D., chief medical officer at Ultragenyx. “The designation is also recognition of the significant clinical benefit observed in the Phase 2 portion of the Orbit study and supports our work to expeditiously bring this investigational therapy to patients who currently have no approved treatment option.”
The FDA’s decision is based on preliminary clinical evidence including the positive 14-month results from the Phase 2 portion of the Orbit study, which demonstrated a rapid and clinically meaningful decrease in fracture rate in patients, and from the completed Phase 2b ASTEROID study. Breakthrough Therapy Designation aims to expedite the development and review of drugs that are intended to treat serious or life-threatening diseases and whose preliminary clinical evidence indicates that the drug may demonstrate substantial improvement on one or more clinically significant endpoints over existing therapies.
Setrusumab was granted Orphan Drug Designation in the United States and EU, rare pediatric disease designation in the United States, and accepted into the European Medicine Agency’s Priority Medicines program (PRIME).
About Osteogenesis Imperfecta (OI)
Osteogenesis Imperfecta (OI) includes a group of genetic disorders impacting bone metabolism. Approximately
About Setrusumab (UX143)
Setrusumab is a fully human monoclonal antibody that inhibits sclerostin, a negative regulator of bone formation. Blocking sclerostin is expected to increase new bone formation, bone mineral density and bone strength in OI. In mouse models of OI, the use of anti-sclerostin antibodies was shown to increase bone formation, improve bone mass to normal levels, and increase bone strength against fracture force testing to normal levels.
In 2019, Mereo BioPharma completed the Phase 2b dose-finding study (ASTEROID) for setrusumab in 112 adults with OI. The ASTEROID study demonstrated treatment with setrusumab resulted in a clear, dose-dependent and statistically significant effect on bone formation and bone density at multiple anatomical sites among adult participants with OI.
Ultragenyx and Mereo BioPharma are collaborating on the development of setrusumab globally based on the collaboration and license agreement between the parties. The companies have developed a comprehensive late-stage program to continue development of setrusumab in pediatric and young adult patients across OI sub-types I, III and IV.
About Ultragenyx
Ultragenyx is a biopharmaceutical company committed to bringing novel therapies to patients for the treatment of serious rare and ultrarare genetic diseases. The company has built a diverse portfolio of approved medicines and treatment candidates aimed at addressing diseases with high unmet medical need and clear biology, for which there are typically no approved therapies treating the underlying disease.
The company is led by a management team experienced in the development and commercialization of rare disease therapeutics. Ultragenyx’s strategy is predicated upon time- and cost-efficient drug development, with the goal of delivering safe and effective therapies to patients with the utmost urgency.
For more information on Ultragenyx, please visit the company's website at: www.ultragenyx.com.
Forward-Looking Statements and Use of Digital Media
Except for the historical information contained herein, the matters set forth in this press release, including statements related to Ultragenyx's expectations and projections regarding its future operating results and financial performance, business plans and objectives for UX143, expectations regarding the tolerability and safety of UX143, and future clinical and regulatory developments for UX143 are forward-looking statements within the meaning of the "safe harbor" provisions of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements involve substantial risks and uncertainties that could cause our clinical development programs, collaboration with third parties, future results, performance or achievements to differ significantly from those expressed or implied by the forward-looking statements. Such risks and uncertainties include, among others, the uncertainty of clinical drug development and unpredictability and lengthy process for obtaining regulatory approvals, the ability of the company and Mereo BioPharma to successfully develop UX143, the risk that fast track or breakthrough designations by the FDA may not lead to faster development or regulatory review or approval process and does not increase the likelihood that UX143 will receive marketing approval, the company’s ability to achieve its projected development goals in its expected timeframes, risks related to adverse side effects, risks related to reliance on third party partners to conduct certain activities on the company’s behalf, the potential for any license or collaboration agreement, including the company’s collaboration agreement with Mereo to be terminated, smaller than anticipated market opportunities for the company’s products and product candidates, manufacturing risks, competition from other therapies or products, and other matters that could affect sufficiency of existing cash, cash equivalents and short-term investments to fund operations, the company’s future operating results and financial performance, the timing of clinical trial activities and reporting results from same, and the availability or commercial potential of Ultragenyx’s products and drug candidates. Ultragenyx undertakes no obligation to update or revise any forward-looking statements.
For a further description of the risks and uncertainties that could cause actual results to differ from those expressed in these forward-looking statements, as well as risks relating to the business of Ultragenyx in general, see Ultragenyx's Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission (SEC) on August 2, 2024, and its subsequent periodic reports filed with the SEC.
In addition to its SEC filings, press releases and public conference calls, Ultragenyx uses its investor relations website and social media outlets to publish important information about the company, including information that may be deemed material to investors, and to comply with its disclosure obligations under Regulation FD. Financial and other information about Ultragenyx is routinely posted and is accessible on Ultragenyx’s Investor Relations website (https://ir.ultragenyx.com/) and LinkedIn website (https://www.linkedin.com/company/ultragenyx-pharmaceutical-inc-/mycompany/).
Contacts
Ultragenyx Pharmaceutical Inc.
Investors
Joshua Higa
+1-415-475-6370
ir@ultragenyx.com
Media
Carolyn Wang
+1-415-225-5050
media@ultragenyx.com
FAQ
What is the Breakthrough Therapy Designation Ultragenyx (RARE) received for setrusumab?
What clinical evidence supported the Breakthrough Therapy Designation for Ultragenyx's (RARE) setrusumab?
What other designations has Ultragenyx (RARE) received for setrusumab?
What is the potential benefit of Breakthrough Therapy Designation for Ultragenyx's (RARE) setrusumab?
