Pfizer has hit the acquisition trail again and is buying FoldRx Pharmaceuticals, in a move that will boost its presence in orphan and rare disease markets.  

Specific financial terms were not disclosed, but the deal consists of an upfront payment and contingent payments if certain milestones are achieved. For the cash, Pfizer will bag investigational compounds to treat conditions caused by protein misfolding, which it says is "increasingly recognised as an underlying cause in many chronic degenerative diseases".  

FoldRx's lead candidate is tafamidis meglumine, an oral, disease-modifying therapy for TTR amyloid polyneuropathy (ATTR-PN), a progressively fatal genetic neurodegenerative disease, for which liver transplant is the only treatment option currently available. Tafamidis has been filed in Europe and the privately-held company says it is currently in communication with the US Food and Drug Administration to define a pathway for submission; the treatment has orphan drug designation on both sides of the Atlantic.

Explaining the rationale behind the deal, Geno Germano, head of Pfizer Specialty Care Business Unit, said it will add "another important component to the growing portfolio of innovative in-line and investigational medicines for orphan and rare diseases" within the company. He added that it will complement "the current and planned future research and clinical development taking place" in the neuroscience disease area.

As for tafamidis, Mr Germano claimed that "we are taking a significant step toward potentially bringing, for the first time, a non-surgical treatment option for underserved patients affected by the deadly disease ATTR-PN". FoldRx chief executive Richard Labaudiniere added that Pfizer's clinical, regulatory and marketing resources, plus its "commitment to the treatment of rare diseases will significantly enhance the ability to pursue the goal of efficiently bringing tafamidis to all patients affected by this devastating neurodegenerative disease".

FoldRx is also working on an early drug discovery programme to identify therapeutic agents for cystic fibrosis, Parkinson's and Huntington's diseases.