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Orphan Drug Designation Granted to Three Active Moieties of INT230-6 for Soft Tissue Sarcoma (STS)

• Post author:Jessica Lynn
• Post published:September 7, 2023
• Post category:Soft Tissue Sarcoma

INT230-6, developed by clinical-stage biotechnology company Intensity Therapeutics (“Intensity”), is an investigational therapy comprising three moieties: cisplatin, SHAO-FA (a penetration enhancing molecule), and vinblastine sulfate. A moiety, in this sense,…

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ICYMI: Gallium Maltolate Earns Orphan Drug Designation for Pediatric Glioblastoma

• Post author:Jessica Lynn
• Post published:August 25, 2023
• Post category:Glioblastoma

  At the moment, there are limited treatment options for glioblastoma, a rare form of brain cancer. While surgery, radiation, chemotherapy, and electric-field therapy are all utilized in the fight…

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MAb-AR20.5 Earns Orphan Drug Designation for Pancreatic Cancer

• Post author:Jessica Lynn
• Post published:August 22, 2023
• Post category:Pancreatic Cancer

  Despite advances in research and the advent of multiple investigational treatments, pancreatic cancer is still incredibly difficult to treat. The five-year survival rate sits at just 12%. Existing therapies…

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NS-089/NCNP-02 Nabs Orphan Drug Designation for DMD

• Post author:Jessica Lynn
• Post published:August 15, 2023
• Post category:Duchenne Muscular Dystrophy

  NS Pharma, Inc., a subsidiary of Nippon Shinyaku Co., recently announced via news release that its therapy NS-089/NCNP-02 was granted Orphan Drug designation from the U.S. Food and Drug…

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Elraglusib Earns Orphan Drug Status for Pancreatic Cancer

• Post author:Jessica Lynn
• Post published:August 15, 2023
• Post category:Pancreatic Cancer

  Even with available therapies, pancreatic cancer is incredibly tough to treat and comes with a poor prognosis. Only 12% of people diagnosed with pancreatic cancer are still alive after…

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Taldefgrobep Alfa Earns EU Orphan Drug Status for Spinal Muscular Atrophy (SMA)

• Post author:Jessica Lynn
• Post published:August 9, 2023
• Post category:Spinal Muscular Atrophy

  Orphan drug designation was first introduced to the European Union in 2000. The designation is granted to therapies intended to treat, diagnose, or prevent a rare, life-threatening, or chronically…

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TYRA-300 Snags Orphan Drug Designation for Achondroplasia

• Post author:Jessica Lynn
• Post published:August 9, 2023
• Post category:Achondroplasia

  Have you ever heard of Orphan Drug designation? This special status was created to incentivize the development of therapies for people with rare conditions (affecting fewer than 200,000 people…

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SAT-3153 Earns Orphan Drug Designation for DMD

• Post author:Jessica Lynn
• Post published:August 6, 2023
• Post category:Duchenne Muscular Dystrophy

  Earlier this year, the FDA granted Accelerated Approval to ELEVIDYS (delandistrogene moxeparvovec-rokl) for the treatmnt of Duchenne muscular dystrophy (DMD) in boys aged 4-5. While this is a stunning…

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SNB-101 Nabs Orphan Drug Status for SCLC

• Post author:Jessica Lynn
• Post published:July 24, 2023
• Post category:Small cell lung cancer/Timely

According to an article from Akosua Mireku of Pharmaceutical Technology, the U.S. Food and Drug Administration recently granted Orphan Drug designation to SNB-101, a potential therapy for people living with…

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ISB 2001 Earns Orphan Drug Designation for the Treatment of Multiple Myeloma

• Post author:Jessica Lynn
• Post published:July 10, 2023
• Post category:Multiple Myeloma/Timely

Orphan Drug designation is granted to therapies that are designed to treat, diagnose, or prevent rare diseases or conditions. In the United States, a rare condition is one affecting fewer…

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FDA Grants ODD to ERAS-801 for Glioblastoma

• Post author:Jessica Lynn
• Post published:June 29, 2023
• Post category:Glioblastoma

  Medical research has yielded a significant number of breakthroughs that have advanced the field and helped people in need. But in the rare disease sphere, it has sometimes been…

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Evorpacept Granted Orphan Drug Designation in Europe for Gastric Cancer

• Post author:Jessica Lynn
• Post published:June 27, 2023
• Post category:gastric cancer

  Throughout the world, it can be difficult to advance research and drug development in the rare disease space. In part, this is because many rare diseases have small population…

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ICYMI: INO-3107 for RRP Earns EC Orphan Drug Designation

• Post author:Jessica Lynn
• Post published:June 23, 2023
• Post category:Recurrent respiratory papillomatosis

  In May 2023, Pharmaceutical Technology reported that INO-3107, a DNA medicine vaccine candidate developed by biotechnology company Inovio Pharmaceuticals (“Inovio”), received Orphan Drug designation from the European Commission. INO-3107…

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RP-A601 Granted Orphan Drug and Fast Track Designations for Rare Cardiomyopathy

• Post author:Jessica Lynn
• Post published:June 20, 2023
• Post category:Arrhythmogenic right ventricular dysplasia/PKP2 arrhythmogenic cardiomyopathy

  Gene therapy has the potential to improve outcomes among individuals across various disease spectrums. In the case of clinical-stage biotechnology company Rocket Pharmaceuticals, the company is working to develop…

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The FDA Grants Orphan Drug Designation to AVB-001 for Mesothelioma

• Post author:Jessica Lynn
• Post published:June 16, 2023
• Post category:Mesothelioma

  Mesothelioma can sometimes be difficult to treat. The cancer can spread to nearby tissue and nerves, and often isn’t diagnosed until later stages. Admittedly, there are a number of…

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Mitazalimab Snags Orphan Drug Designation for Pancreatic Cancer

• Post author:Jessica Lynn
• Post published:June 12, 2023
• Post category:Pancreatic Cancer/Timely

Although the 5-year survival rate for pancreatic cancer has slightly risen (from 11% to 12%), there is still much work to be done. Pancreatic cancer can be difficult to treat.…

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Obeticholic Acid – Bezafibrate Combination Granted Orphan Drug Designation for Primary Biliary Cholangitis (PBC)

• Post author:Jessica Lynn
• Post published:June 7, 2023
• Post category:Primary Biliary Cholangitis

  In mid-May 2023, Pharmaceutical Technology reported that a new combination treatment had earned Orphan Drug designation. This combo? Obeticholic acid (OCA) and bezafibrate. Together, they represent a potential therapeutic…

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PYX-201 Granted Orphan Drug Designation for the Treatment of Pancreatic Cancer

• Post author:Jessica Lynn
• Post published:May 18, 2023
• Post category:Pancreatic Cancer/Timely

Pyxis Oncology is driven to improve the lives of people living with cancer through the development of innovative immunotherapy and antibody drug conjugate (ADJ) therapies. Their treatment PYX-201, a novel…

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ICYMI: NTLA-5001 Granted Orphan Drug Designation for the Treatment of AML

• Post author:Jessica Lynn
• Post published:April 27, 2023
• Post category:Acute Myeloid Leukemia

  The passage of the Orphan Drug Act was an important step in creating an environment that supports rare disease research and drug development. This Act created Orphan Drug designation,…

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FDA Grants Orphan Drug Designation to Oxypurinol for ADPKD

• Post author:Jessica Lynn
• Post published:April 26, 2023
• Post category:Autosomal Dominant Polycystic Kidney Disease

  In February 2023, I spoke with Dr. Allen Davidoff of XORTX Therapeutics Inc. (“XORTX”) about the company’s commitment to developing therapies for people living with progressive kidney disease. We…

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FDA Grants Orphan Drug Designation to Ganaxolone for Lennox-Gastaut Syndrome

• Post author:Jessica Lynn
• Post published:April 5, 2023
• Post category:Lennox-Gastaut syndrome

  Prior to the end of March 2023, ganaxolone had received six separate Orphan Drug designations from the FDA; more recently, as shared in a news release from commercial-stage pharmaceutical…

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ISB 1442 for Multiple Myeloma Earns Orphan Drug Designation

• Post author:Jessica Lynn
• Post published:March 22, 2023
• Post category:Multiple Myeloma

Cases of multiple myeloma seem to be rising across the country and the globe. New and effective therapeutic interventions have the potential to significantly benefit this patient population. Ichnos Sciences…

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EP0042 for AML Earns Orphan Drug Designation

• Post author:Jessica Lynn
• Post published:March 16, 2023
• Post category:Acute Myeloid Leukemia

  The Orphan Drug Act was passed with a goal in mind: to incentivize the development of therapeutics for orphan drugs. These drugs are used for patients with rare conditions,…

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YB-200 Granted Orphan Drug Designation for HCC

• Post author:Jessica Lynn
• Post published:March 7, 2023
• Post category:Hepatocellular Carcinoma/Timely

It can be difficult to spur interest in rare disease drug development. Forty years ago, the Orphan Drug Act passed as a way to incentivize drug development within this space.…

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Ketarx for Rett Syndrome Earns Orphan Drug Designation

• Post author:Jessica Lynn
• Post published:February 13, 2023
• Post category:Rett Syndrome

In the United States, Orphan Drug designation is granted by the FDA to drugs focused on the treatment, prevention, or diagnosis of rare conditions; these are those affecting fewer than…

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