Latest news

• NICE backs Amicus Therapeutics’ Galafold

  and Shire's Replagal (agalsidase alfa).

• GSK gene therapy tops CHMP recommendations

  The drug could offer a patient-friendly alternative to injectable Fabry therapies such as Sanofi/Genzyme's Fabrazyme (agalsidase beta) and Shire's Replagal (agalsidase alfa).

• Sanofi gains accelerated review for rare disease drug

  But even though catering for a small patient population (around 10, 000 people are thought to have the disorder globally), the firm has competition in Shire's Replagal (agalsidase alfa).

• AbbVie coaxes Shire into discussions with sweetened offer

  as Elaprase (idursulfase) for Hunter syndrome and Replagal (agalsidase alfa) for Fabry disease.

• The moving parts of orphan drug development

  The company already had its own orphan brands such as Replagal (agalsidase alfa) for Fabry disease, Hunter's syndrome treatment Elaprase (idursulfase) and Vpriv (velaglucerase alfa) for Gaucher disease, when it