How to Navigate the FDA Landscape for an Orphan Drug
Rare diseases are a public health priority. FDA has launched several programs and a final guidance to address the unmet need for orphan drugs.
Rare diseases are a public health priority. FDA has launched several programs and a final guidance to address the unmet need for orphan drugs.
先天性副腎過形成症(CAH)は、世界中でおよそ400,000人の患者さんがいます。CAHに対する現在の治療では、標準的な治療レジメンはなく、さまざまな汎用グルココルチコイドステロイド(米国ではヒドロコルチゾン、デキサメタゾン、プレドニゾロン、プレドニゾンなど)が使用されています。The cortisol deficiency and over-production of male sex hormones caused by CAH can lead to increased mortality, infertility, and sexual development … Continued
In 2020, the US FDA approved Risdiplam (Evrysdi®) as the first orally administered drug to treat SMA in patients ≥2 months old, followed by the European Medicine Agency. Risdiplam addresses the underlying cause of SMA: a reduced amount of survival motor neuron (SMN) protein.
Everolimus (Afinitor®) was recently approved as adjunctive therapy for tuberous sclerosis complex (TSC)-associated partial seizures in ages 2 and older. Unlike many other therapies for treating TSC-associated seizures, everolimus addresses the underlying pathophysiology of TSC. As TSC-associated seizures can also affect children aged between 6 months and 2 years, a modeling and simulation approach that … Continued
Pharmacylics and J&J sought to bring ibrutinib, its new tyrosine kinase inhibitor therapy targeting rare B-cell malignancies to market. The company leveraged FDA’s accelerated approval program; it was one of the first to be awarded breakthrough status by the agency. Ibrutinib is susceptible to interactions with a strong inhibitor and inducer of CYP3A4 enzymes, thus … Continued