Shire lifted by rare disease approvals
pharmafile | January 26, 2015 | News story | Sales and Marketing | HGT-2310, Hunter syndrome, SHP609, Shire, fast track, idursulfase-IT, rare disease
Shire’s investigational drug for a rare neurological disease has been accepted into the FDA’s fast-track approval process.
The drug, SHP609, (idursulfase-IT, also known as HGT-2310) received an FDA Fast Track designation as a treatment for neurocognitive decline associated with Hunter syndrome, a rare genetic disorder.
SHP609 is a new formulation of Shire’s existing treatment for Hunter syndrome, Elaprase (idursulfase). It is designed to be used alongside Elaprase and administered directly into the cerebrospinal fluid.
Hunter Syndrome (also known as mucopolysaccharidosis II or MPSII) is a severely debilitating rare disease that affects 1 in 162,000 babies born. It is caused by the lack of an enzyme, and can lead to severe health problems and early mortality.
The FDA’s Fast Track program is designed to help companies to get drugs reviewed more quickly, especially drugs that “address serious or life-threatening conditions and that demonstrate the potential to address unmet medical needs”.
Being given the designation boosts a company’s chances of getting a propriety review for a drug by granting more meetings with FDA regulators, if the clinical data is robust.
In Shire’s case it is currently enrolling patients in its Phase II/III pivotal trial to look at the effect of SHP609 children with Hunter syndrome and early cognitive impairment – who being treated with Elaprase.
“This is not only the first treatment being investigated to address the significant unmet need of slowing the cognitive decline in MPS II patients, but also the furthest a program for enzyme replacement has ever progressed,” says Philip Vickers, head of research and development at Shire.
“This Fast Track designation is further recognition of the critical need to develop new, effective therapy options for patients with Hunter syndrome with cognitive impairment.”
The Irish company also received good news from the FDA after NPS Pharma, the company Shire paid $5.2 billion for earlier this month, gained an FDA approval for Natpara (parathyroid hormone) as an additional treatment for hypocalcemia in patients with hypoparathyroidism who cannot take standard treatments.
Because of a known potential risk of bone tumours (osteosarcoma), Natpara will be made available in the US through a Risk Evaluation and Mitigation Strategy (REMS) Program.
In response to the NPS Pharma decision on Natpara, Shire chief executive Flemming Ornskov, comments: “The Natpara label is in line with our expectations, and we believe this approval further validates Shire’s decision to acquire NPS Pharma, which is an excellent strategic fit allowing us to leverage our market expertise, core capabilities in rare disease patient management, and global footprint.
“We look forward to combining our strengths with NPS Pharma to launch NATPARA in the US after the expected close of the transaction in Q1 of this year.”
Lilian Anekwe
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