According to a press release from April 8, 2021, a partnership between Project ALS and the ALS Association has allowed for the development of a Phase 3 clinical trial. Two…
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…
Financial Burdens in Rare Disease In 2019, 20% of all adults in the United States have reported that they had large medical bills that were unexpected. 18% currently had medical…
Lyme disease, and other tick-borne illnesses, can cause intense and prolonged symptoms. In fact, some patients experience chronic symptoms for a number of years following the initial bite. However,…
On March 29, 2021, global biopharmaceutical company Bristol Myers Squibb ("BMS") shared that its type II variation application for Opdivo (nivolumab) was validated by the European Medicines Agency (EMA).…
In a recent press release, clinical-stage gene therapy company Rocket Pharmaceuticals, Inc. ("Rocket") shared that its investigational gene therapy candidate, RP-L201, received Priority Medicines (PRIME) designation from the European…
During the Society of Gynecological Oncology 2021 Virtual Annual Meeting on Women’s Cancer, researchers presented data from the Phase 2 OVARIO clinical trial. According to Cancer Network, the trial evaluated…
Rocket Pharmaceuticals, Inc. ("Rocket") has dedicated its mission to developing gene therapy solutions for rare childhood conditions. Recently, the company shared the publication of preliminary data from a Phase…
Genomic testing is a great way to learn not just about your genes, but how your genes impact your health. In support of the Phase 2 CRESTONE clinical trial, a…
In a press release from late February 2021, biopharmaceutical company Retrotope shared that its lead drug candidate, RT001, received Rare Pediatric Disease designation for the treatment of patients with Friedreich's…
How much should medication cost, and are these treatments cost-effective and accessible for patients? In a press release, biopharmaceutical company Aurinia Pharmaceuticals, Inc. ("Aurinia") shared that an independent review performed…
Since its inception, life sciences company Solid Biosciences Inc. has been working to develop treatment options for patients with Duchenne muscular dystrophy (DMD). According to a recent press release, the…
In a recent press release, biopharmaceutical company Swedish Orphan Biovitrum AB ("Sobi") shared that the first patient was dosed in a Phase 3 clinical trial evaluating avatrombopag for pediatric patients…
Together, biopharmaceutical company Sanofi and biotechnology company Regeneron Pharmaceuticals, Inc. ("Regeneron") have been working to develop Libtayo (cemiplimab) for patients with advanced cervical cancer. According to a press release,…
Pamrevlumab is a first-in-class antibody that treats Duchenne muscular dystrophy (DMD) by inhibiting the effects and activity of connective tissue disorder growth factor (CTGF). It is being developed by FibroGen,…
Fast track designation is granted by the FDA. If a drug developer receives this designation, they begin a process to develop and expedite review for drugs or biologics which treat…
In a recent press release, the Ivy Brain Tumor Center, a nonprofit translational science initiative, shared that the first patient was treated in a Phase 0 clinical trial. The clinical…
At the end of February 2021, BridgeBio Pharma Inc. ("BridgeBio") and its affiliate Origin Biosciences, Inc. ("Origin") shared that its treatment, NULIBRY (fosdenopterin) for Injection, received FDA approval for patients…
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…
In a recent press release, biopharmaceutical company Salarius Pharmaceuticals, Inc. ("Salarius") shared that it had initiated an expansion stage of a Phase 1/2 clinical trial. Within the trial and the…
In a press release from March 3, 2021, biotechnology company BioMarin Pharmaceutical Inc. ("BioMarin") shared that it had completed full enrollment for a Phase 2 clinical trial evaluating vosoritide for…
At the beginning of March 2021, gene therapy company AVROBIO, Inc. shared that its investigational gene therapy, AVR-RD-04, was given Orphan Drug designation by the European Commission (EC). This treatment…
Lysogene has just announced that the FDA has approved their Investigational New Drug Application (IND) for a therapy for GM1 gangliosidosis, a rare pediatric condition. The investigational treatment is a…
During the virtual XVIII International Conference on Duchenne and Becker Muscular Dystrophy, specialty pharmaceutical company Italfarmaco Group ("Italfarmaco") shared updates regarding various Givinostat clinical development programs. The treatment is designed…
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