As reported by Oncology Nursing News, gunagratinib (ICP-192) was recently granted Orphan Drug designation by the FDA. Altogether, this treatment is being evaluated as a potential treatment for patients…
Rare Pediatric Disease designation is a status granted by the FDA to drugs or biologics intended to treat rare diseases (affecting under 200,000 Americans) in patients aged 18 or younger.…
Prior to 2021, there were no FDA-approved treatments for patients with type 1 plasminogen deficiency, or hypoplasminogenemia. As a result, there was an urgent need to fill this unmet need…
In a news release from June 2, 2021, biopharmaceutical company Soleno Therapeutics, Inc. ("Soleno") shared an exciting update. The company's investigational treatment, DCCR (diazoxide choline) Extended-Release tablets, received Orphan Drug…
In early June 2021, SPG302, an investigational therapy for patients with amyotrophic lateral sclerosis (ALS), received Orphan Drug designation from the FDA. According to ALS News Today, the treatment…
According to a recent news release from biopharmaceutical company Arena Pharmaceuticals, Inc. ("Arena"), the company's investigational therapy, etrasimod, received Orphan Drug designation from the FDA. Currently, Arena is evaluating etrasimod…
According to Yahoo Finance, immuno-oncology company Surface Oncology ("Surface") recently received Orphan Drug designation for its leading therapeutic candidate SRF617. Altogether, this therapy is designed to treat patients with pancreatic…
In a recent press release, clinical stage biotech and biopharmaceutical company Biom Therapeutics ("Biom") shared that its drug candidate BIO017 was granted Orphan Drug designation. The treatment, designed for patients…
At the very end of March 2021, specialty pharmaceuticals company JCR Pharmaceuticals Co., Ltd. ("JCR") shared that its investigational drug candidate JR-171 received Orphan Drug designation from the European Medicines…
NORD (the National Organization for Rare Disorders), through PR Newswire, recently announced the results of a new study that reveals the total number of generics, orphan products, and biosimilars currently…
In a press release from early March 2021, cell therapy company CiRC Biosciences, Inc. ("CiRC") shared that its chemically induced photoreceptor-like cells (CiPCs) received Orphan Drug designation from the FDA.…
We seldom see articles about multiple system atrophy (MSA also called Shy-Drager syndrome). The disease is extremely rare and currently, there is no effective treatment. But according to a recent…
First formed and launched within the UPstart incubator at the University of Pennsylvania's Penn Center for Innovation (PCI), biopharmaceutical company Linnaeus Therapeutics, Inc. ("Linnaeus") is now making strides in the…
A report, commissioned by the National Organization for Rare Disorders and released by IQVIA, has revealed that the majority of orphan drugs, 79%, treat only rare diseases. Despite this fact,…
On March 11, 2021, global biopharmaceutical company Alkermes plc shared that its investigational drug candidate nemvaleukin alfa received Orphan Drug designation from the FDA. The treatment is designed for patients…
In a recent press release, biotech company AltruBio Inc. shared that its therapy neihulizumab (AbGn-168H) received Fast Track designation for the treatment of patients with steroid refractory acute graft-versus-host disease…
The FDA's Orphan Drug designation offers a special status to drugs or biologics designed to treat rare conditions. In the United States, a rare disease is one that impacts less…
Just over a week ago, drug discovery and development company Rescindo Therapeutics Inc. ("Rescindo") shared that its drug candidate RSC-57 received both Orphan Drug and Rare Pediatric Disease designations. The…
According to European Pharmaceutical Review, results from a Phase 1/2a clinical trial support the use of AstroRx for patients with amyotrophic lateral sclerosis (ALS). The clinical trial, headed by Kadimastem…
In a recent press release, pharmaceutical company Acer Therapeutics, Inc. ("Acer") announced the publication of data on celiprolol for patients with COL3A1-positive vascular Ehlers-Danlos Syndrome (vEDS). The article, published in the European…
In a recent press release, biopharmaceutical company Acceleron Pharma, Inc. ("Acceleron") announced that its investigational drug candidate sotatercept received Orphan designation from the European Commission (EC) for the treatment of…
On November 20, 2020, biopharmaceutical company RedHill Biopharma Ltd. announced the initiation of a Phase 3 clinical trial to study the orally administered RHB-204. The drug is designed to treat…
According to a story from PR Newswire, the biopharmaceutical company reVision Therapeutics, Inc. has just announced that its experimental therapy REV-0100 has received both Rare Pediatric Disease designation and Orphan…
Recently, gene therapy company Taysha Gene Therapies ("Taysha") announced that its gene therapy candidate, TSHA-102, received both Orphan Drug and Rare Pediatric Disease designations from the FDA. TSHA-102, delivered via…
In December 2019, the FDA approved Ervebo, the first-ever Ebola vaccine. However, despite preventative measures, there still was never any actually approved treatment options - until now. Recently, the FDA…
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