Codexis announces FDA orphan drug and rare paediatric disease designations for CDX-6512 for the treatment of homocystinuria

24 January 2022 - Codexis today announced that the U.S. FDA has granted the company orphan drug designation for CDX-6512 for ...

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Orphan drugs: privilege of "fictitious" additional benefit not justified

12 January 2022 - Drugs for orphan diseases should also go through a regular benefit assessment procedure when they enter the ...

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U.S. FDA approves Immix Biopharma rare paediatric disease designation for IMX-110 as a treatment for life-threatening paediatric cancer

3 January 2021 - Immix Biopharma announced today that the U.S. FDA has granted rare paediatric disease designation for IMX-110 for ...

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New hope offered to rare disease patients

14 December 2021 - Early this month, the world's first therapy to treat spinal muscular atrophy, a rare genetic disorder ...

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Canada's national strategy for drugs for rare diseases needs a holistic approach

8 December 2021 - International report summarises drugs for rare disease best practices and recommendations. ...

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FDA takes new steps aimed at advancing development of individualised medicines to treat genetic diseases

7 December 2021 - Today, the FDA is issuing a draft guidance to provide recommendations for managing the administration of individualised ...

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Fortress Biotech, Cyprium Therapeutics and Sentynl Therapeutics announce the initiation of rolling submission of a new drug application for CUTX-101, copper histidinate, for treatment of Menkes disease

7 December 2021 -  Cyprium Therapeutics with support from its licensing partner Sentynl Therapeutics today announced the initiation of a ...

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It’s time to better support the three million Canadians with a rare disease

25 November 2021 - After finding out her private health insurer would no longer pay for a drug helping with ...

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FDA approves first drug to improve growth in children with most common form of dwarfism

19 November 2021 - Today, the U.S. Food and Drug Administration approved Voxzogo (vosoritide) injection to improve growth in children five ...

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NICE does not recommend elosulfase alfa for NHS use following evaluation of new evidence

12 November 2021 - NICE has today published draft guidance for public consultation which does not recommend elosulfase alfa (also called ...

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Drugs against rare diseases: Libmeldy gene therapy shows additional benefits - Zolgensma cell therapy, on the other hand, does not

4 November 2021 - The Federal Joint Committee (G-BA) today assessed the additional benefit of two active ingredients against very rare, ...

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FDA approves innovative treatment for paediatric patients with congenital athymia

8 October 2021 - Today, the U.S. FDA approved Rethymic for the treatment of paediatric patients with congenital athymia, a rare ...

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U.S. FDA approves Livmarli (maralixibat) as the first and only approved medication for the treatment of cholestatic pruritus in patients with Alagille syndrome one year of age and older

29 September 2021 - Mirum Pharmaceuticals today announced that the U.S. FDA has approved Livmarli (maralixibat) oral solution for the treatment ...

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Aduhelm backlash threatens to reverse progress in FDA’s reviews of rare and ultra-rare disease drugs

14 September 2021 - The FDA’s approval of Aduhelm to treat Alzheimer’s disease has unleashed criticism about the decision and ...

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Albireo receives UK MHRA approval of Bylvay (odevixibat)

8 September 2021 - Bylvay now approved in U.S., EU and UK as first drug treatment for patients with progressive familial intrahepatic ...

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