Core Viewpoint - Klotho Neurosciences, Inc. has received Orphan Drug Designation from the FDA for its gene therapy product KLTO-202, aimed at treating ALS, highlighting the company's commitment to developing innovative treatments for rare neurodegenerative diseases [1][3][5]. Group 1: Company Overview - Klotho Neurosciences, Inc. focuses on gene and cell therapies for neurodegenerative and aging-related diseases, utilizing a patented form of the "anti-aging" human Klotho gene [7]. - The company is developing KLTO-202, which targets motor neuron diseases and muscular dystrophies through a muscle-specific promoter and aims to deliver therapeutic concentrations of the s-KL protein [6][7]. Group 2: Orphan Drug Designation - The FDA's Orphan Drug Designation is granted to treatments for rare diseases affecting fewer than 200,000 people in the U.S., providing incentives such as tax credits and seven years of market exclusivity [2]. - The designation for KLTO-202 validates the scientific approach of Klotho Neurosciences in addressing ALS, a disease with significant unmet medical needs [2][3]. Group 3: ALS Context - ALS, also known as Lou Gehrig's disease, affects fewer than 200,000 people in the U.S., with approximately 5,000 new cases diagnosed annually [4]. - The disease is characterized by motor neuron damage, leading to severe physical decline and is universally fatal [3][4]. Group 4: Development Progress - Klotho Neurosciences has completed proof of concept studies in animal models and is initiating manufacturing of KLTO-202, with plans for further discussions with the FDA and EMA regarding development [5].

In an ongoing Phase 1 trial published in Nature Medicine, MB-101 was well-tolerated and 50% of patients achieved stable disease or better with two partial responses and two complete responses lasting 7.5 and 66+ months, respectively Preclinical data support a novel combination of MB-101 (IL13Rα2‐targeted CAR-T cell therapy) and MB-108 (HSV-1 oncolytic virus) to optimize clinical results FDA previously granted Orphan Drug Designation to Mustang for MB-108 for the treatment of malignant glioma WORCESTER, Mas ...

Jaguar Health, Inc. (NASDAQ: JAGX) What's Different????? 3 NASDAQ:JAGX • The classic definition of insanity: Doing the same thing over and over again and expecting a different result • Equally crazy — changing the approach and expecting to get the same results Overview – May 2025 Forward-Looking Statements This presentation contains "forward-looking statements" within the meaning of Section 21E of the Securities Exchange Act of 1934, as amended. All statements other than statements of historical facts conta ...

Submission Seeks Approval of GTx-104 in the Treatment of Patients with aneurysmal Subarachnoid Hemorrhage (aSAH) Comprehensive Data Package Includes Positive Results from Phase 3 STRIVE-ON Safety Trial of GTx-104 Submission Has the Potential to Trigger Exercise of up to $7.6 million in Gross Proceeds from 2023 Financing Warrants Tied to FDA Acceptance of NDA for Review PRINCETON, N.J., June 25, 2025 (GLOBE NEWSWIRE) -- Grace Therapeutics, Inc. (Nasdaq: GRCE) (Grace Therapeutics or the Company), a late-stage ...

U.S. FDA grants Orphan Drug Designation to Deramiocel for the treatment of Becker Muscular Dystrophy, broadening Capricor’s focus in neuromuscular diseasesCapricor remains on track for the August 31, 2025, PDUFA date for Deramiocel in Duchenne Muscular Dystrophy following successful FDA Pre-License Inspection SAN DIEGO, June 17, 2025 (GLOBE NEWSWIRE) -- Capricor Therapeutics (NASDAQ: CAPR), a biotechnology company developing transformative cell and exosome-based therapeutics for the treatment of rare diseas ...

SOUTH SAN FRANSCISCO, Calif., June 17, 2025 (GLOBE NEWSWIRE) -- CERo Therapeutics Holdings, Inc., (Nasdaq: CERO) (“CERo” or the “Company”) an innovative immunotherapy company seeking to advance the next generation of engineered T cell therapeutics that deploy phagocytic mechanisms, announces that the U.S. Food and Drug Administration (FDA) has granted CERo’s Orphan Drug Designation (ODD) for the company's lead drug candidate CER-1236, for the treatment of acute myeloid leukemia (AML). CER-1236 is an innovat ...

EMA Orphan Drug Designation (ODD) builds on U.S. Food & Drug Administration (FDA) ODD and Fast Track Designation, underscoring the urgent need for new treatment optionsThe multiple regulatory designations were granted following strong positive results from a global randomized Phase 1b/2 study in first-line HCC demonstrating superior outcomes for amezalpat combination therapy across multiple study endpoints, including overall survival in both the entire population and key subpopulations, when compared to sta ...

DUBLIN, June 05, 2025 (GLOBE NEWSWIRE) -- Avadel Pharmaceuticals plc (Nasdaq: AVDL), a biopharmaceutical company focused on transforming medicines to transform lives, announced today that LUMRYZTM has been granted Orphan Drug Designation (ODD) from the U.S. Food & Drug Administration (FDA) for the treatment of Idiopathic Hypersomnia (IH). Specifically, ODD was granted based on the plausible hypothesis that LUMRYZ may be clinically superior to the same drug(s) already approved for the same indication, becaus ...

Key Takeaways Sanofi's rilzabrutinib receives FDA orphan drug designation for SCD, its fourth such indication. Preclinical data show rilzabrutinib reduced vaso-occlusion and inflammation in SCD mouse models. Rilzabrutinib is also under FDA review for ITP, with a decision expected by August 29, 2025.Sanofi (SNY) announced that the FDA has granted orphan drug designation to its investigational BTK inhibitor, rilzabrutinib, for sickle cell disease (SCD). The candidate exerts its effects through multi-immune ...

Core Viewpoint - The FDA has granted orphan drug designation to rilzabrutinib for sickle cell disease, highlighting its potential to address unmet medical needs in rare diseases [1][2]. Company Overview - Sanofi is an R&D driven biopharma company focused on improving lives through innovative medicines and vaccines, with a commitment to addressing urgent healthcare challenges [7]. Product Information - Rilzabrutinib is a novel, advanced, oral, reversible Bruton's tyrosine kinase (BTK) inhibitor that aims to restore immune balance through multi-immune modulation [5]. - The drug has received multiple orphan drug designations, including for immune thrombocytopenia (ITP), warm autoimmune hemolytic anemia (wAIHA), and IgG4-related disease (IgG4-RD) [2][8]. Disease Context - Sickle cell disease affects over 100,000 people in the US, predominantly impacting African American and Hispanic populations, leading to severe pain and reduced life expectancy [6]. - The disease is characterized by misshapen red blood cells that block blood flow, causing various health complications [6]. Regulatory Status - Rilzabrutinib is currently under regulatory review in the US, EU, and China for its potential use in ITP, with a target action date for FDA decision set for August 29, 2025 [3][8].