Ipsen announces U.S. FDA priority review for palovarotene new drug application in patients with fibrodysplasia ossificans progressiva following resubmission

29 June 2022 - Ipsen today announced that the U.S. FDA has accepted for priority review its resubmitted new drug ...

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Nexviadyme (avalglucosidase alfa) approved by European Commission as a potential new standard of care for the treatment of Pompe disease

28 June 2022 - Approved for the treatment of the full spectrum of both late-onset Pompe disease and infantile-onset Pompe disease. ...

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TLV receives assignments for medicines for rare diseases

27 June 2022 - The Government has commissioned the Swedish Dental and Pharmaceutical Benefits Agency (TLV) to analyse and make ...

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Rhythm Pharmaceuticals announces FDA approval of Imcivree (setmelanotide) for use in patients with Bardet-Biedl syndrome

16 June 2022 - Approval based on Phase 3 trial results that demonstrated statistically significant reductions in weight and hunger in ...

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EFPIA-EURORDIS joint statement on patient access to medicines for rare diseases

15 June 2022 - Today marks the publication of the EFPIA-EURORDIS joint statement on patient access to medicines for rare diseases.  ...

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Cancer, rare disorder advocates call for more radical change after damning PHARMAC review

2 June 2022 - Radical action is needed urgently to tackle the delays patients face in getting the latest drugs for ...

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CHMP recommends approval of Xenpozyme (olipudase alfa), the first and only treatment for ASMD

20 May 2022 - Recommendation based on positive results from two clinical trials in which Xenpozyme provided improvement across multiple non-CNS ...

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Selumetinib for treating symptomatic and inoperable plexiform neurofibromas associated with type 1 neurofibromatosis in children aged 3 and over

5 May 2022 - NICE has published evidence based recommendations on the use of selumetinib (Koselugo) for the treatment of children ...

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Pliant Therapeutics receives FDA fast track designation for PLN-74809 for the treatment of idiopathic pulmonary fibrosis

3 May 2022 -  Pliant Therapeutics announced today that PLN-74809, its oral, dual-selective αvß6/αvß1 integrin inhibitor, has received fast track ...

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Orphalan announces FDA approval of Cuvrior for the treatment of adult patients with stable Wilson’s disease who are de-coppered and tolerant to penicillamine

2 May 2022 - Orphalan announces FDA approval of Cuvrior for the treatment of adult patients with stable Wilson’s disease ...

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Ontario family forced to fight for life-saving drug highlights need for national rare disease strategy

21 April 2022 - Beth Vanstone doesn’t want anyone else to have to fight for access to life saving medications. ...

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ICER publishes white paper evaluating reforms to orphan drug development, pricing and coverage

7 April 2022 - The white paper presents an analysis of the potential risks and benefits of reforms seeking to ...

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Statement from Rare Disorders NZ re Minister Little's comments on PHARMAC funding

31 March 2022 - Rare Disorders NZ is extremely frustrated to hear the Minister of Health this week dismiss the call ...

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ABPI response to England Rare Diseases Action Plan

28 February 2022 - England's Rare Disease Action plan is published today with details of how to improve diagnosis, care and ...

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Protalix BioTherapeutics and Chiesi Global Rare Diseases announce the submission of a marketing authorisation application to the European Medicines Agency for PRX-102 for the treatment of Fabry disease

24 February 2022 - Protalix BioTherapeutics and Chiesi Global Rare Diseases today announced the submission of a marketing authorisation application via ...

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