In the past, medical research has shown that the STAT3 protein plays a role in regulating inflammatory responses throughout the body; it also plays a role in cell proliferation,…
The FDA has just provided AB001 the Orphan Drug designation for both acute myeloid leukemia (AML) and pancreatic cancer. This small molecule was developed by Vopec Pharmaceuticals and Agastiya Biotech.…
In Europe, Orphan Drug designation is granted to drugs or biologics intended to treat, diagnose, or prevent rare, life-threatening, or chronically debilitating conditions. For the European Union (EU), a “rare”…
Earlier this month, I wrote about how efzitimod, a therapy for systemic sclerosis, received Orphan Drug designation from the FDA. Now, another therapy – this one from Gesynta Pharma (“Gesynta”)…
Have you ever heard of Orphan Drug designation before? The FDA grants Orphan Drug designation to drugs or biologics intended to treat, diagnose, or prevent rare diseases or conditions. In America,…
The European Commission (EC) has recently granted its Orphan Drug designation to AR101, a PKCβ inhibitor for the treatment of Ehlers-Danlos syndrome (EDS). This decision was based off of a…
Silmitasertib, an investigative therapy for biliary tract cancer (BTC), has been awarded Orphan Drug Designation by the FDA. Researchers and the regulatory agency are hopeful that this therapy could become…
The FDA has just announced that they have granted Orphan Drug Designation to an investigative treatment called eplontersen. This therapy is being studied as a treatment option for transthyretin (ATTR)…
Karyopharm Therapeutics has just announced that Eltanexor, their investigative therapy for myelodysplastic syndromes (MDS), has been given Orphan Drug Designation by the FDA. This designation means that the FDA believes…
Jaguar Health and Napo EU S.p.A have just announced that crofelemer, an investigative therapy for short bowel syndrome (SBS) has been granted Orphan Drug Designation by the EMA. Thanks to…
In the United States, Orphan Drug designation is granted to drugs or biologics intended to treat, diagnose, or prevent rare diseases. These are defined as conditions affecting under 200,000 Americans.…
Mereo BioPharma Group has just announced that they have received Orphan Drug Designation from the FDA for their new treatment for alpha-1 antitrypsin deficiency (A1AD) called alvelestat. This therapy is…
To best address the needs of patients with rare diseases and rare cancers, additional treatment options are necessary. But developing these treatment options can be time-consuming and sometimes difficult.…
According to a press release published on Globe Newswire, the biopharmaceutical company Saniona has announced recently that its investigational therapy Tesomet has recently earned Orphan Drug designation from the US…
According to a story from Market Watch, the gene therapy company AVROBIO, Inc. has recently announced that its investigational gene therapy candidate AVR-RD-04 has earned Orphan Drug designation from the…
The Orphan Drug Act originated in 1983 as an incentive for pharmaceutical companies who worked on developing treatments for rare disease. Since rare diseases are well, rare, choosing to work…
As reported in BioSpace; today, Biocryst Pharmaceuticals has been given a boost to further their work to explore the merits of a potential oral treatment for PNH. The U.S. FDA…
In a recent press release, biopharmaceutical company Regulus Therapeutics, Inc. announced that their drug candidate, RGLS4326, received Orphan Drug Designation for the treatment of autosomal dominant polycystic kidney disease…
This past week, biopharmaceutical company Kiniksa Pharmaceuticals announced that their drug, rilonacept, received Orphan Drug Designation from the FDA. Following positive data from the RHAPSODY Phase 3 clinical trial,…
A gene therapy for the treatment of GM1 gangliosidosis was granted Orphan Drug Designation in April. PBGM01 seeks to treat the most severe form of this condition - infantile. Read…
In June 2019, Dicerna Pharmaceuticals submitted a clinical trial application for their DCR-A1AT therapy and expects to treat the first patient with A1AD-related liver disease near the end of…
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