Shire has signed a deal worth up to $440 million with US biotechnology firm Amicus Therapeutics which sees the UK drugmaker get access to three investigational compounds that treat rare genetic disorders.

Shire has licensed the ex-US rights to Amicus’ Amigal (migalastat) for Fabry disease, which is in Phase II and Plicera (isofagomine) for Gaucher disease, also in Phase II. The deal also covers AT2220 (deoxynojirimycin) for Pompe disease, which is still in Phase I.

Cashwise, Amicus will receive an upfront fee of $50 million and up to $150 million more if certain research milestones are met. If the drugs get to market, Amicus will receive up to $240 million in sales milestone payments, plus double-digit royalties, and retain the US rights. The companies will split the cost of development and commercialisation of the compounds.

Shire’s chief executive Matthew Emmens said that Amicus’ “pharmacological chaperone compounds” have the potential to be “an excellent addition to our current enzyme replacement therapy business”, which includes Replagal (agalsidase alfa) for the treatment of Fabry disease and Elaprase (idursulfase) for Hunter syndrome. The company also has GA-GCB (velaglucerase alfa) in Phase III for Gaucher disease, said Mr Emmens who noted that the Amicus deal gives Shire an entry into the market for Pompe disease.

Specialising in the area of Fabre, Gaucher and Pompe disease has principally been Genzyme Corp’s territory and these disorders have proved to be very lucrative for the US firm. Shire will be hoping to grab a bigger slice of that market down the line.