Everolimus approved to treat rare tumour disease
pharmafile | September 8, 2011 | News story | Sales and Marketing | Novartis, SEGA, Votubia, everolimus
Novartis has gained EU approval for everolimus to be used to treat a rare non-cancerous tumour.
Everolimus is already marketed as Afinitor in cancer and as Certican for organ rejection, and will now be known as Votubia to treat subependymal giant cell astrocytoma (SEGA).
These non-cancerous brain tumours arise because of an underlying condition, tuberous sclerosis complex (TSC) which affects an estimated one to two million people worldwide and is associated with a variety of resulting disorders including seizures, swelling in the brain, developmental delays and skin lesions.
TSC is a genetic disorder that may cause non-cancerous tumours to form in vital organs and can affect many different parts of the body, most commonly the brain and kidney. SEGAs, or non-cancerous brain tumours, occur in up to 20% of patients with TSC.
The approval of the oral drug represents a significant advance for patients with the condition, as brain surgery was the only treatment option until now.
The drug is licensed to treat adults and children from the age of three who require therapeutic intervention but are not amenable to surgery.
The approval is based on a prospective, open-label, single-arm, Phase II study of 28 patients which showed that 78% of patients experienced a reduction of 30% or greater in the size of their largest SEGA and 33% (9 of 27) experienced a reduction of 50% or greater at six months relative to baseline. A further placebo-controlled phase III study was recently presented, and met its primary endpoint of SEGA response rate.
“The approval of Votubia in the European Union means that for the first time, patients living with SEGA associated with tuberous sclerosis complex will have an effective therapeutic option,” said Hervé Hoppenot, president of Novartis Oncology.
Everolimus targets mTOR, a protein that acts as an important regulator of tumour cell division, blood vessel growth and cell metabolism.
Novartis’ Hervé Hoppenot added: “This milestone serves as another example of the potential of mTOR inhibition with everolimus and we remain steadfast in our commitment to studying other manifestations of TSC.”
The SEGA approval comes just three days after another new approval for the drug. Afinitor was approved for the treatment of unresectable or metastatic, well- or moderately-differentiated neuroendocrine tumors (NET) of pancreatic origin in adults with progressive disease.
In commercial terms, the approvals represent a success for Novartis’ pursuit of multiple and diverse licences for everolimus, which could help it reach total annual sales in excess of $1 billion.
Andrew McConaghie
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 …
