PHARMAC calls for applications for medicines for rare disorders

21 November 2022 - PHARMAC is calling for applications for medicines for rare disorders.  ...

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Asfotase alfa for the treatment of patients with paediatric-onset hypophosphatasia

4 November 2022 - The Department of Health and Social Care has asked NICE to review the guidance on the ...

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Kiwis with rare disorders want faster progress on 'life or death' health strategy

6 November 2022 - For Samantha Lenik, being diagnosed with Pompe​ disease after seven years of symptoms was a “double-edged ...

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Rare disease endpoint advancement pilot program

3 October 2022 - FDA is establishing a Rare Disease Endpoint Advancement (RDEA) Pilot Program to support novel efficacy endpoint development ...

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Agios receives positive CHMP opinion for Pyrukynd (mitapivat) for the treatment of pyruvate kinase deficiency in adult patients

16 September 2022 - Agios expects a decision on the marketing authorisation application by the European Commission within 67 days of ...

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Chiesi Global Rare Diseases announces FDA acceptance of BLA filing for velmanase alfa for the proposed treatment of alfa mannosidosis

12 September 2022 - FDA grants priority review designation with PDUFA date in the first half of 2023. ...

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Rare cancer sufferers fight to get drugs on PBS

9 September 2022 - People with rare cancers are being forced to sell their homes, raid their superannuation and fundraise ...

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The FDA needs to be more flexible in assessing treatments for rare diseases, like the one that seemed to help my son

7 September 2022 - Every time I read about clinical trials testing possible treatments for rare diseases, I think of ...

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Australia among world's best in treating debilitating and often fatal muscle disorder

4 September - Australia has become one of the first countries in the world to fund a new, next-generation treatment ...

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FDA approves first treatment for acid sphingomyelinase deficiency, a rare genetic disease

31 August 2022 - Today, the US FDA approved Xenpozyme (Olipudase alfa) for intravenous infusion in paediatric and adult patients with ...

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Patient registries: EMA officials highlight opportunities in orphan drug development

25 August 2022 - Officials from the EMA tout the benefits of patient registries to support regulatory decision making for ...

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‘No magic bullet’: For drug makers and the FDA, clinical trials on ultra rare diseases pose thorny challenges

26 July 2022 - Walker Burger is beside himself with worry. ...

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Alnylam receives positive CHMP opinion for vutrisiran for the treatment of hereditary transthyretin-mediated amyloidosis in adult patients with stage 1 or stage 2 polyneuropathy

22 July 2022 - Positive opinion based on HELIOS-A Phase 3 study. ...

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Velmanase alfa for the treatment of alfa mannosidosis

15 July 2022 - The Department of Health and Social Care has asked NICE to produce guidance on using velmanase alfa ...

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US FDA awards rare paediatric disease designation to paxalisib for AT/RT, a rare form of childhood brain cancer

6 July 2022 - Kazia Therapeutics is pleased to announce that the U.S. FDA has awarded rare paediatric disease designation ...

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