PhRMA Reports 450 Medicines in Development for Rare Diseases
PhRMA reports 452 new medicines are in development for rare diseases, including genetic disorders, neurological conditions, infectious diseases, and autoimmune disorders.
The
(PhRMA) reports that biopharmaceutical research companies are developing 452 new medicines for rare diseases, including genetic disorders, neurological conditions, infectious diseases, and autoimmune disorders. According to FDA, in the last five years, one third of all new drug approvals were for rare diseases.
The 452 medicines and vaccines in development for rare diseases, all in human clinical trials or under review by FDA, include 105 for cancer, 85 for genetic disorders, 65 for blood cancers, and 32 for neurological disorders.
Source:
Drug Solutions Podcast: A Closer Look at mRNA in Oncology and Vaccines
April 30th 2024In this episode fo the Drug Solutions Podcast, etherna’s vice-president of Technology and Innovation, Stefaan De Koker, discusses the merits and challenges of using mRNA as the foundation for therapeutics in oncology as well as for vaccines.
Understanding the Variability in Bioburden Test Results in Biomanufacturing
May 7th 2025This article explores the impact of test volume, microbial distribution, and dilution errors on bioburden testing variability. It presents statistical approaches to estimate percentage error and discusses strategies to optimize microbial enumeration techniques in biopharmaceutical quality control.
Drug Solutions Podcast: Gliding Through the Ins and Outs of the Pharma Supply Chain
November 14th 2023In this episode of the Drug Solutions podcast, Jill Murphy, former editor, speaks with Bourji Mourad, partnership director at ThermoSafe, about the supply chain in the pharmaceutical industry, specifically related to packaging, pharma air freight, and the pressure on suppliers with post-COVID-19 changes on delivery.
Intertek to Expand UK GMP Facility with 6000 Square Feet of Lab and Office Space
May 7th 2025The company said the expansion is in response to globally rising demand for inhaled biologics, which offer distinct advantages in route of administration, but can be challenging products for developers.
