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Small molecule drug shows promise in rare disease
In a Phase II trial, the small-molecule drug CBL-514 demonstrated significant reduction in lipoma size and pain improvement in Dercum's disease, a rare disorder.
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Rare diseases
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CHMP meeting highlights – May 2023
The Committee for Medicinal Products for Human Use (CHMP) recommended two new medicines for approval in its May meeting, including a neurosteroid for epileptic seizures.
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$1.7b acquisition to develop rare haematology therapy
When CTI BioPharma is acquired by Swedish Orphan Biovitrum AB (Sobi), the companies intend to advance an oral kinase inhibitor treatment for a rare haematological disease.
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Takeda to build £600m plasma therapy manufacturing site
Takeda’s largest manufacturing capacity expansion investment in Japan will fund the build of a new facility for plasma-derived therapies.
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CHMP recommends first pegylated enzyme for Fabry disease
A PEGylated enzyme replacement therapy for Fabry disease has received recommendation for a marketing authorisation from the European Medicines Agency (EMA)'s human medicines committee.
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mRNA non-vaccine therapeutics to generate $2 billion by 2028
Driven by the approval of pipeline agents, mRNA non-vaccine therapeutics are set to reach $2 billion in sales by 2028, says GlobalData.
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Orphan drug sector continues to experience rapid growth
According to a new report, the orphan drug market is growing more than twice as fast as the non-orphan market and, by 2026, orphan drug sales will account for 20 percent of all prescription drug sales.
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UK bioscience companies form Rare Disease Industry Group
The BIA is committed ensuring patient access to treatments for rare and very rare conditions, sometimes referred to as orphan and ultra-orphan medicines...
