The FDA recently informed Muscular Dystrophy News that it has granted orphan drug designation to the experimental drug ISX9-CPC, a product of the IPS Heart company located in Houston, Texas.…
The FDA is taking significant steps to update and streamline its regulatory approach to rare diseases, according to a recent article from BioCentury.com. Recognizing the unique challenges faced by patients,…
An article over at Genetic Engineering & Biotechnology News reports that Pfizer has withdrawn its sickle cell disease medication Oxbryta after the European Medicines Agency (EMA) disclosed the deaths of…
The U.S. Food and Drug Administration has announced a public meeting set for Wednesday, October 16, 2024 to discuss the creation of a Rare Disease Innovation Hub. The meeting is…
Rare diseases are challenging. Not just for those affected, but for researchers, physicians, and drug developers. Rare diseases may affect small populations. Many are poorly understood and lack adequate awareness.…
As reported in Biopharma Dive, the FDA’s decision to strengthen its authority involving laboratory developed tests (LDTs) has drawn considerable objections from various healthcare groups. However, the Agency has held…
In July 2023, the U.S. FDA granted Orphan Drug designation to ABM-1310, an investigational treatment option for people living with malignant gliomas such as glioblastoma. Now, reports clinical-stage biopharmaceutical…
The Orphan Drug designation was created to aid and hasten the development of drugs created for the treatment, diagnosis, or prevention of rare diseases. When the FDA grants this designation,…
Bristol Myers Squibb (BMS) has been developing luspatercept-aamt - marketed as Reblozyl - as a treatment for anemia in non-transfusion dependent (NTD) beta thalassemia. They submitted a supplemental biologics license…
Despite undergoing clinical development, many drugs never receive approval from the FDA. Recently, this happened to a treatment for growth hormone deficiency (GHD). The medication, titled somatrogon and owned by…
The Critical Path Institute (C-Path), formed in 2005, is a nonprofit, independent (public/private) partnership. The Institute evolved from the FDA’s 2004 White Paper known as the Critical Path Report and…
Fabry disease patients may have another treatment option soon, according to a press release published in BioSpace. Protalix Biotherapeutics and Chiesi Global Rare Diseases have provided an update on the…
According to an article submitted to StatNews by members of the Barth Syndrome Foundation, the FDA approved the drug Aduhelm in accordance with its accelerated approval program. Although the treatment…
The FDA has just announced their approval of zanubrutinib (Brukinsa) as a therapeutic option for Waldenström's macroglobulinemia. This approval was given based on the ASPEN Phase 3 clinical trial, and…
As of June 7, 2021, an injectable treatment is now available for patients aged 1 month+ with paroxysmal nocturnal hemoglobinuria. The FDA recently approved Ultomiris (ravulizumab-cwvz) for these patients. Previously,…
The FDA has recently accepted NeoImmuneTech's Investigational New Drug (IND) application for NT-17, a treatment for progressive multifocal leukoencephalopathy (PML). Now that the application has been cleared, the biopharmaceutical company…
The medical community has been aware for some time now that patient involvement in his or her treatment is a critical part of therapeutic development. Biopharmaceutical companies acknowledge their obligation…
In a late December 2020 press release, biopharmaceutical company Cerecor Inc. (“Cerecor”) announced the FDA acceptance of an Investigational New Drug Application (IND) for CERC-007. The human monoclonal antibody (mAb)…
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…
COVID-19’s tentacles have invaded the New Drug Applications (NDAs) for 2020. A recent article in Fierce Pharma reported that the FDA has been especially vigilant in scrutinizing NDAs this year,…
The FDA has officially approved remdesivir for the treatment of adults and children (over the age of 12 and weighing at least 40 kg) who have been hospitalized for COVID-19.…
This week, the US FDA announced that they approved the drug Nucala (mepolizumab) to treat patients with hypereosinophilic syndrome, a rare chronic blood disease which causes organ damage. Nucala is…
In a recent press release, clinical-stage pharmaceutical company Crinetics Pharmaceuticals announced that their investigational drug candidate, CRN04777, received Rare Pediatric Disease designation from the FDA. The therapy is designed…
According to biotechnology Gilead Sciences, the FDA rejected a new drug application (NDA) for filgotinib. The drug, designed to treat patients with moderate to severe rheumatoid arthritis (RA), is known…
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,…
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