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…
According to a February 25 press release, Amondys 45 (casimersen), a therapeutic option for patients with Duchenne muscular dystrophy (DMD), recently became FDA-approved. The therapy is specifically designed for patients…
On February 18, 2021, specialty pharmaceutical company Kyowa Kirin Co., Ltd. ("Kyowa Kirin") shared that a Phase 2 clinical trial evaluating KHK4083 had reached its primary endpoint. KHK4083 is an…
In 2007, biotechnology company Seagen Inc. and pharmaceutical company Astellas Pharma Inc. ("Astellas") collaborated to develop enfortumab vedotin. Now, in 2021, the pair submitted two supplemental Biologics License Applications (sBLAs)…
During clinical development, treatments are sometimes first evaluated in healthy volunteers. This is the route that Insmed Incorporated ("Insmed") took with treprostinil palmitil inhalation powder (TPIP). Now, following positive topline…
Recently, botanical pharmaceutical company Devonian Health Group Inc. ("Devonian") has been evaluating its product thykamine for patients with mild-to-moderate atopic dermatitis in a Phase 2 clinical trial. In a…
Biotechnology company Stealth Biotherapeutics ("Stealth") has made the company's mission to develop and commercialize treatments for patients with diseases characterized by mitochondrial dysfunction. One leading treatment in the company's…
From February 25-27, the Americas Committee for Treatment and Research in Multiple Sclerosis will run its ACRTIMS Forum 2021, according to BioSpace. At this forum, Clene Inc. will present interim…
A Phase 3 study of upadacitinib, an ulcerative colitis treatment, met both its primary and secondary endpoints, according to an announcement from AbbVie. The trial, titled U-ACCOMPLISH, saw a third…
Recently, clinical stage biopharmaceutical company Immunic, Inc. ("Immunic") has been working to develop potential treatment options for patients with primary sclerosing cholangitis (PSC), a rare condition for which no…
According to Targeted Oncology, the first patient was dosed in a Phase 1 clinical trial evaluating NBTXR3, an investigational tumor-agnostic radio enhancer, for patients with esophageal cancer. The trial will…
While gene therapy is a promising field, there are some associated risks: unwanted immune reactions, infections, or whether the therapy could lead to the development of other conditions, like cancer.…
In early February, global biopharmaceutical company Sanofi and biotechnology company Regeneron Pharmaceuticals, Inc. ("Regeneron") shared that its dually developed drug Libtayo (cemiplimab-rwlc) recently received FDA approval. Now, Libtayo stands as…
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