FDA holds up Pfizer’s rare disease drug tafamidis
pharmafile | June 19, 2012 | News story | Research and Development, Sales and Marketing | FDA, Pfizer, rare disease, tafamidis
The FDA has sent Pfizer a Complete Response Letter about its rare disease drug tafamidis meglumine.
The US regulator is asking Pfizer for a second clinical study to establish ‘substantial evidence of effectiveness’ before it will approve the drug.
The agency has also asked for additional information on the data within the new drug application for tafamidis.
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 in Europe and has been available on the continent under the brand name Vyndaqel since November.
The FDA had granted priority review to tafamidis in February to speed up the regulatory process, but its passage through the FDA will now be delayed.
Pfizer said in a statement that it would “work with the FDA to address the content of the letter”.
Dr Yvonne Greenstreet, senior VP and head of medicines development group for Pfizer’s speciality care business unit, said: “TTR-FAP is a relentless and debilitating disease. We understand the urgent need within the patient community and stand firmly behind this innovative medicine.
“It is our intention to request a meeting as soon as possible with the agency in order to discuss a potential path forward.”
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.
FoldRx, a drug discovery firm that specialises in treatments for diseases caused by ‘protein misfolding’ originally developed the treatment. Pfizer bought the firm last year, acquiring tafamidis in the process.
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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