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Drug Development

A total of 77 posts are filed under Drug Development
U.S. Food and Drug Administration Orphan Drug Designations in December 2017
The following is a list of FDA Orphan Drug designation made in December 2017… Continue Reading
Savara Advances Pipeline of Inhaled Therapeutics for Rare Lung Diseases
PAP and NTM are two rare lung conditions with different pathologies. PAP is an autoimmune condition while NTM is caused by a bacterial… Continue Reading
FDA Approves Expended Indication for Procysbi to Include Children with Nephropathic Cystinosis
Rare Daily Staff The U.S. Food and Drug Administration approved an expansion to the indication for Horizon Pharma’s Procysbi to include… Continue Reading
FDA Say It Needs an Additional Three Months to Review BioMarin’s PKU Drug
Rare Daily Staff BioMarin Pharmaceutical said that the U.S. Food and Drug Administration will require additional time to complete its review… Continue Reading
Why the Microbiome May Provide a Novel Approach to Treating Certain Rare Diseases
The microbiome has become an area of great interest among drug developers as a way to treat diseases. Synlogic, which is developing a new… Continue Reading
FDA approves Addmedica’s Siklos for Kids with Sickle Cell Anemia
Rare Daily Staff The Food and Drug Administration approved Addmedica’s Siklos to reduce the frequency of painful crises and the need for… Continue Reading
Shire’s Experimental Therapy for MPS II Fails in Phase 2/3 Study
Rare Daily Staff Rare disease drugmaker Shire said that its experimental therapy SHP609 for MPS II, also known as Hunter Syndrome, failed to… Continue Reading