Neurenati Therapeutics Inc., a biotech company focused on rare pediatric diseases, is pleased to announce that the U.S. FDA has granted both Orphan Drug Designation (ODD) and Rare Pediatric Disease Designation (RPDD) to NEU-001, a novel combination therapy utilizing a neurotrophic growth factor, for the treatment of Hirschsprung disease (HD). “We hope for a cure, one child at a time.” -- Maxime Ranger Ranger, CEO of Neurenati Therapeutics
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When it comes to navigating the complexities of FDA regulations, the stakes are incredibly high and the margin for error is slim. Choosing the right partner can make all the difference. At KLA Breakthrough Consulting, we specialize in navigating the intricate landscape of FDA regulations to ensure the most efficient path to market. We craft individual regulatory strategies designed to result in Accelerated Approval. Early on, for our clients with rare disease indications, we secure crucial designations like Orphan Drug Designation (ODD) and Rare Pediatric Disease Designation (RPD). We also help determine whether an INTERACT meeting is appropriate, and which questions are most likely to result in an FDA response. As your therapeutic moves closer to clinical trials, we provide GAP analyses and Pre-IND meetings, as well as INDs and eCTD publishing services. We specialize in Fast Track, RMAT, and Breakthrough designations for drug developers, which enable the most efficient path to NDA/BLA and marketing approval. Boasting an industry-leading success rate of over 95% on ODD and RPD, our approach starts with a meticulous pre-contract data analysis, followed by a tailor-made strategy to meet each Sponsor’s unique needs. Our mission is to be more than just consultants; we aim to be dedicated partners in giving your therapeutic its best chance for fast, efficient FDA approval. Driven by an unwavering commitment to both scientific rigor and client relationships, we offer transparent, collaborative, and personalized services that set us apart in the industry. ? 700+ FDA Submissions ? 300+ FDA Orphan Drug Requests ? 100+ FDA Pediatric Rare Disease Applications ? 50+ Fast Track, Breakthrough, RMAT
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https://klabreakthroughconsulting.com/
KLA Breakthrough Consulting的外部链接
- 所属行业
- 生物技术研究
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- 2-10 人
- 总部
- US and EU
- 类型
- 自有
- 创立
- 2019
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- Rare Disease、High Unmet Medical Need、orphan drugs、FDA Fast Track 、FDA Breakthrough Designation、FDA Meetings、eCTD Publishing和IND
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KLA Breakthrough Consulting员工
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Sandra Kirsch Heibel
Science and Regulatory Affairs expert seeking opportunities to continue supporting development of rare disease therapies through consulting, teaching…
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Elena Gostishcheva
Aspiring Regulatory Affairs Professional | RA Intern at KLA Breakthrough Consulting
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Jason Farmer
PharmD
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Kelly Austin PhD, PPM
Regulatory Specialist ? We provide high-quality regulatory affairs consulting that is tailored to Sponsors' individual needs, and designed to get…
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KLA Breakthrough Consulting转发了
Regulatory Specialist ? We provide high-quality regulatory affairs consulting that is tailored to Sponsors' individual needs, and designed to get therapeutics to market as quickly as possible.
''The rare pediatric disease priority review voucher program remains in its sunsetting state – the current authority only allows for granting of such vouchers if the drug was designated as a drug for a rare pediatric disease not later than December 20, 2024, and for which a “rare pediatric disease product application” is approved not later than September 30, 2026. In the meantime, the Pink Sheet?confirmed?that FDA was continuing to review rare pediatric disease designation requests.?However, we were not aware of any actual grants of rare pediatric disease designations past the December 20, 2024, deadline – until recently. We wish to applaud FDA for continuing to grant these designations, and we congratulate the sponsors in their efforts.?While the program is currently closed without a qualifying ticket for entry, these sponsors have set themselves up well to take advantage of the program should Congress reauthorize it and bring these zombie designations back to life.?We remain hopeful that this will happen in the near future.''
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Regulatory Specialist ? We provide high-quality regulatory affairs consulting that is tailored to Sponsors' individual needs, and designed to get therapeutics to market as quickly as possible.
Hack Rare: Engaging the Next Generation of Rare Disease Innovators This is the team of Harvard University undergraduates who participated in last year’s Harvard Rare Disease Hackathon, sponsored by the National Organization for Rare Disorders (NORD). Their team won “Most Novel Solution” at last year’s Hackathon for their medical device model for Charcot Marie Tooth Disease: the CMT4J pressure-reducing customizable boot. ''The Harvard Rare Disease Hackathon sponsored by NORD provided us with the opportunity to think critically, collaborate with peers from diverse backgrounds, and provide hope for new innovation to a disease that, to date, has no cure and few supportive therapies. The event demonstrates how cultivating an environment of cross-field innovation can be integral in the fight for rare disease innovation.'' This hybrid event allowed students from around the country to collaborate, test, and pitch technology-based solutions to today’s rare disease challenges.?The Hackathon is taking place again this year, on March 1-2, 2025. Link to register in the comments.
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The FDA has granted orphan drug designation to the systemic radiopharmaceutical 225Ac-satoreotide (SSO110) as a potential therapeutic option for patients with small cell lung cancer. “Receiving orphan drug designation for 225Ac-satoreotide is a recognition of its potential as a treatment option for patients with SCLC and an important regulatory milestone for Ariceum,” Manfred Ruediger, chief executive officer at Ariceum Therapeutics, stated in a news release. “The FDA’s orphan drug designation will support our objective to accelerate the development of satoreotide through human trials to provide a potentially life-saving therapy to patients with limited alternatives.”
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KLA Breakthrough Consulting转发了
Regulatory Specialist ? We provide high-quality regulatory affairs consulting that is tailored to Sponsors' individual needs, and designed to get therapeutics to market as quickly as possible.
FDA Issues Draft Guidance on Accelerated Approval: A Substantial Evidentiary and Procedural Overhaul to this High-Profile Pathway ''FDA’s new accelerated approval guidance is a must read. The discussion provides a wide-ranging and much needed update on FDA policies and interpretation of its accelerated approval authorities.'' Link to full article in the comments below
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KLA Breakthrough Consulting转发了
Regulatory Specialist ? We provide high-quality regulatory affairs consulting that is tailored to Sponsors' individual needs, and designed to get therapeutics to market as quickly as possible.
FDA approves drug to prevent or reduce the frequency of bleeding episodes for patients with hemophilia A with inhibitors or hemophilia B with inhibitors "The development of inhibitors remains the most serious treatment-related complication for people living with hemophilia. For patients with inhibitors, especially in hemophilia B, their hemophilia may remain poorly controlled and pose a life-threatening risk," said Shapiro, MD, CEO and co-medical director at the Hemophilia & Thrombosis Center, Inc. "The approval of Alhemo??– a first-of-its-kind, prophylaxis, subcutaneous injection pen for adults and children 12 years and older with hemophilia A and B with inhibitors – provides a much-needed alternative to the current standard of care in hemophilia B with inhibitors, while offering patients with hemophilia A with inhibitors more treatment options, ultimately providing more patients with inhibitors the opportunity to personalize their care and address current treatment gaps."
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Regulatory Specialist ? We provide high-quality regulatory affairs consulting that is tailored to Sponsors' individual needs, and designed to get therapeutics to market as quickly as possible.
FDA Approves First Mesenchymal Stromal Cell Therapy to Treat Steroid-refractory Acute Graft-versus-host Disease Ryoncil is the first FDA-approved MSC therapy. It contains MSCs, which are a type of cell that can have various roles in the body and can differentiate into multiple other types of cells. These MSCs are isolated from the bone marrow of healthy adult human donors. “Today’s decision marks an important milestone in the use of innovative cell-based therapies to treat life-threatening diseases with devastating impacts on patients, including children,” said Peter Marks, M.D., Ph.D., director of the FDA’s Center for Biologics Evaluation and Research (CBER). “This first mesenchymal stromal cell therapy approval demonstrates the FDA’s commitment to supporting the development of safe and effective products that could improve the quality of life for patients with symptoms that are unresponsive to other therapies.” The FDA granted approval of Ryoncil to Mesoblast Limited, Inc.
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Regulatory Specialist ? We provide high-quality regulatory affairs consulting that is tailored to Sponsors' individual needs, and designed to get therapeutics to market as quickly as possible.
The FDA has approved cosibelimab-ipdl (Unloxcyt) from Checkpoint Therapeutics Inc for the treatment of adults with metastatic or locally advanced cutaneous squamous cell carcinoma (CSCC) who are not candidates for curative surgery or radiation. "Today’s FDA approval of Unloxcyt – the first marketing approval for our company – is a significant milestone both for Checkpoint and for patients with advanced CSCC,” James Oliviero, CFA, president and chief executive officer of Checkpoint Therapeutics, stated in a news release.
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Regulatory Specialist ? We provide high-quality regulatory affairs consulting that is tailored to Sponsors' individual needs, and designed to get therapeutics to market as quickly as possible.
FDA approves Crenessity (crinecerfont) to be used together with glucocorticoids (steroids) to control androgen (a testosterone-like hormone) levels in adults and pediatric patients 4 years of age and older with classic congenital adrenal hyperplasia (CAH). Today’s approval provides an important advance for patients with classic congenital adrenal hyperplasia and highlights the FDA’s continued commitment to advancing effective and safe treatments for rare diseases,” said Theresa Kehoe, MD, director of the Division of General Endocrinology in the FDA’s Center for Drug Evaluation and Research. “The FDA will continue working with patients, drug companies and health care providers to address the unmet medical needs of the rare disease community.” The FDA granted the approval of Crenessity to Neurocrine Biosciences, Inc.
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KLA Breakthrough Consulting转发了
Regulatory Specialist ? We provide high-quality regulatory affairs consulting that is tailored to Sponsors' individual needs, and designed to get therapeutics to market as quickly as possible.
''If the program were to sunset completely, as currently scheduled, the impact on the development of drugs for rare pediatric diseases would be difficult to overstate. Although FDA cannot currently award rare pediatric disease priority review vouchers to any applications not currently designated as a drug for a rare pediatric disease, there is no provision that prevents FDA from continuing to grant such designations. We would strongly urge companies to continue to request, and FDA to continue to grant, such designations in the hopes that Congress can accomplish what it seemed ready to do on a bipartisan basis just a few days ago.?In the meantime, we encourage all stakeholders to keep the pressure on to get this done as soon as possible.''