FDA accepts Pfizer rare disease drug for review
pharmafile | February 15, 2012 | News story | Sales and Marketing | EMA, FDA, FoldRx, Pfizer, tafamidis meglumine
The FDA has accepted for review Pfizer’s new drug to treat a rare neurodegenerative disease.
Pfizer is seeking a licence for tafamidis meglumine to treat Transthyretin Familial Amyloid Polyneuropathy (TTR-FAP), a rare, progressive and fatal neurodegenerative disease that affects around 8,000 patients worldwide.
The drug is already approved and has been available in Europe under the brand name Vyndaqel since November.
The FDA has granted priority review to tafamidis, and said that a decision on the drug could be reached as early as June. A priority review is granted to treatments that have the potential to offer significant improvement in treatment, or where no adequate therapy exists.
FoldRx, a drug discovery firm that specialises in treatments for diseases caused by ‘protein misfolding’ originally developed the drug. Pfizer bought the firm last year, acquiring tafamidis in the process.
The drug works by targeting misfolded proteins, which occur when proteins fail to achieve the correct three-dimensional structure. It stabilises transthyretin and thereby inhibits amyloid formation and progression of the transthyretin amyloidosis.
Following 18 months of treatment in one pivotal clinical study, more patients treated with Pfizer’s drug met the definition of response with respect to the Neuropathy Impairment Score of the Lower Limb (NISLL), than patients on placebo.
Less deterioration of neurologic function as measured by other endpoints and improved nutritional status were also observed in patients treated with tafamidis.
Patients with TTR-FAP experience a significantly diminished quality of life due to symptoms including polyneuropathy characterised by sensory loss, pain and weakness in the lower limbs.
As the disease progresses, patients often lose the ability to walk, needing wheelchair assistance, and eventually become bedridden and unable to care for themselves.
The disease typically occurs during active adult years with onset usually in a patient’s 30s, followed by disease progression that may reach the terminal stage in just 10 years on average.
Ben Adams
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