FDA approves Novartis’ Afinitor
pharmafile | November 1, 2010 | News story | Sales and Marketing | Afinitor, Novartis, subependymal giant cell astrocytomas, tuberous sclerosis
Novartis’ drug Afinitor has been approved by US authorities as a first line treatment for patients with benign brain tumours associated with the genetic disorder tuberous sclerosis (TS).
Previously, patients with growing subependymal giant cell astrocytomas (SEGA), which primarily affect children and adolescents, faced the prospect of brain surgery.
TS affects between 25,000 and 40,000 people in the US and SEGAs occur in up to 20% of patients.
Although trials of Afinitor have yet to confirm the drug’s clinical benefit, US patients will now have access to it because TS is such an area of unmet medical need.
Hervé Hoppenot, president of Novartis Oncology, said: “Today’s FDA decision is an important milestone for the children and adults living with SEGA associated with tuberous sclerosis. We are committed to furthering research for patients with TS and will continue to work towards addressing their unmet medical needs.”
The FDA’s accelerated approval of Afinitor is based on an open-label, single-arm, 28-patient study conducted by Cincinnati Children’s Hospital Medical Center.
In this nearly one-third of patients there experienced a reduction of 50% or more in the size of their largest SEGA at six months relative to baseline, with no new tumours reported.
A phase III study across ten countries is now underway to evaluate the findings of the Cincinnati study. EXIST-1 compares Afinitor to placebo and endpoints include SEGA response, seizure rate and skin lesion response rate.
The Tuberous Sclerosis Alliance welcomed the FDA’s decision.
“SEGAs can be challenging for individuals with tuberous sclerosis and for the whole family, which is why we are encouraged to see ongoing research and new treatment options” says vice president and chief scientific officer Vicky Whittemore.
Applications for an indication in SEGA have also been filed with the European Medicines Agency and the Swiss Agency for Therapeutic Products and if approved in the EU, the drug’s trade name would be Votubia, Novartis says.
The drug is already indicated for renal cancer, but in the summer the UK’s cost-effectiveness body NICE rejected Afinitor (everolimus) for NHS use on grounds of cost.
Adam Hill
Related Content
Novartis receives SMC approval for early breast cancer treatment
Novartis has announced that its treatment for early breast cancer, Kisqali (ribociclib), has received approval …
Novartis candidate for Sjögren’s disease presents positive results
Novartis has reported positive results from two phase 3 clinical trials – NEPTUNUS-1 and NEPTUNUS-2 …
First malaria medicine for infants under 4.5kg receives approval
Coartem (artemether-lumefantrine) Baby, or Riamet, has been approved by Swissmedic as the first malaria medicine …
