PTC Therapeutics has achieved a significant milestone with the U.S. Food and Drug Administration’s (FDA) approval of SEPHIENCE™ (sepiapterin), marking the first and only therapy approved for broad treatment of…
June 2025 marked a month of significant progress in cancer care, as the FDA approved several new therapies and treatment combinations across a range of cancer types. As reported by…
Fujifilm Corporation’s investigational therapy FF-10832 has received Orphan Drug Designation from the U.S. Food and Drug Administration (FDA) for the treatment of biliary tract cancer, according to a recent announcement…
Global biotechnology leader CSL recently announced the FDA’s approval of ANDEMBRY, a monthly subcutaneous (under-the-skin) self-injection. The treatment was recently FDA approved for the prevention of hereditary angioedema (HAE) attacks…
On Monday, February 12 the Food and Drug Administration approved Eohilia, a budesonide corticosteroid oral suspension for patients aged 11 years and older with eosinophilic esophagitis (EoE) patients in the…
A recent article on Yahoo Finance, reports that Alnylam Pharmaceutical Inc.’s RNAi therapeutic, Amvuttra, has been approved by the FDA to treat polyneuropathy of hereditary transthyretin-mediated (hATTR) amyloidosis. Amvuttra (Vutrisiran)…
Medscape has recently reported on a new supplemental approval from the FDA for setmelanotide, which is a treatment for obesity associated with Bardet-Biedl syndrome (BBS). Now, BBS patients who are…
For the last 52 years, individuals with Wilson disease have been relying, largely, on the same treatment options. These options, designed to remove copper from the body, include Cuprimine (penicillamine).…
Lennox-Gastaut syndrome (LGS) is a rare, severe form of epilepsy that is often resistant to anti-seizure medications. This means that patients still experience epileptic episodes despite treatment. Because of this…
According to a recent press release, the FDA has recently granted approval to Vijoice - also known under the generic name alpelisib - for the treatment of PIK3CA-related overgrowth spectrum…
Until recently, patients with cyclin-dependent kinase-like 5 (CDKL5) deficiency disorder (CDD) did not have an FDA-approved treatment option. This all changed when the regulatory agency granted approval to ZTALMY, also…
It's officially Rare Disease Week, which is being celebrated from February 22nd until March 2nd. Every year, we take this week to to recognize, support, raise awareness, and advocate for…
According to a recent article from Pharmacy Times, Octapharma’s cutaquig has been approved by the FDA to treat patients who are 2 years of age or older with primary humoral…
It is extremely important to get the right medications into the hands of patients who need them. Recently, the FDA helped do this for pediatric patients (aged 2+) with primary…
According to a news release from biopharmaceutical company Clearside Biomedical, Inc. ("Clearside") and eye health business Bausch + Lomb, a revolutionary treatment called XIPERE (triamcinolone acetonide injectable suspension) was recently…
In the past, there have not been any available treatments to tackle cholestatic pruritus, or a severe and unrelenting itch, for patients with liver diseases such as Alagille syndrome. However,…
The FDA has recently approved a new diagnostic tool to help determine which IDH1-mutated cholangiocarcinoma patients may be a good fit for the novel IDH1 inhibitor called ivosidenib. The new…
According to a recent article at Targeted Oncology, the FDA has approved abatacept (Orencia) under priority review for preventing moderate to severe acute graft versus host disease (GvHD). The patients…
Accessing treatments can be difficult, especially if one is impacted by a rare disease. In fact, only 5% of rare diseases have an FDA-approved treatment. Because of this, many patients…
According to a recent article in Biospace, patients with advanced systemic mastocytosis (SM) can turn to a newly FDA-approved treatment by Blueprint Medicines called Ayvakit (avapritinib). The drug is the…
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…
The FDA has recently approved DANYELZA for the treatment of neuroblastoma in pediatric patients over the age of one and adult patients affected by refractory or relapsed neuroblastoma. This approval…
An article recently appeared in Health News by NPR describing progeria, an extremely rare disease. There are only twenty people in the United States diagnosed with progeria. The disease causes…
According to Ankylosing Spondylitis News, a new treatment, Hulio, has been approved by the FDA. It is a treatment for ankylosing spondylitis and other inflammatory disorders. Although it has been…
According to an article published by Biospace, X4 Pharmaceuticals, based in Cambridge, Massachusetts, has recently announced successful phase two results in clinical trials for their experimental drug X4P-001. The drug…
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