LYON Sanofi's Genzyme unit has received positive feedback from U.S. doctors after the recent launch of multiple sclerosis pill Aubagio and is moving toward refiling its FDA application for MS drug Lemtrada, senior executives said on Friday.
The two drugs are the pillars of Sanofi's entry into the multiple sclerosis market as the French drugmaker seeks new growth areas to offset the loss of patents protecting its former blockbuster drugs.
Genzyme aims to become a major player in the growing multiple sclerosis market and challenge its Cambridge, Massachusetts rival Biogen Idec, which sells two MS drug and is developing BG-12, a new pill that could become one of the leading MS drugs on the market if approved.
Genzyme, which launched Aubagio in the U.S. on October 1, is presenting detailed data on the pill from a previously published study at a medical congress in Lyon, France.
"We are 11 days in, so it's pretty early, but reports are extremely positive," Genzyme's head of MS Bill Sibold told Reuters.
The data presented in Lyon confirmed that a daily dose of the treatment reduced the rate of relapse by 36 percent compared with a placebo in more than 1,169 patients with relapsing forms of multiple sclerosis.
Aubagio is less effective than rival pills but has milder side effects and analysts say it could be popular among newly diagnosed patients or those who want to switch from interferons, injectable drugs that are commonly used to treat the chronic disease.
Although Aubagio's lack of serious side effects is likely to encourage doctors' prescriptions, its modest efficacy may squeeze its take-up once BG-12 comes to the market, Deutsche Bank analysts said in a note to investors.
Biogen said on Friday that its confidence for BG-12 was bolstered by analyzing combined data from the two late-stage studies, and suggested that it could get U.S. marketing approval by the end of the year.
Genzyme also said it is making progress toward refiling Lemtrada in the United States, after the Food and Drug Administration asked the company to change the presentation of its application to clarify the data.
"We are on track to satisfy the questions of navigability from the FDA," said Michael Panzara, therapeutic area head for multiple sclerosis and immune diseases at Genzyme. "We are also having interactions with regulators around the world which are very favorable."
Lemtrada, which is given via an intravenous drip for five days and then for three days a year later, has the potential to re-program the immune system and could potentially change the long-term progression of the disease.
But analysts say doctors could limit prescriptions of the drug, if approved, to more severe or high-risk patients because of its side effects, which include infections and autoimmune disorders.
As a result, expectations for Lemtrada have remained low. The drug is expected generate sales of just $454.8 million in 2017 compared with $2.95 billion for Biogen Idec's injectable drug Tysabri, according to Thomson Reuters Pharma forecasts.
Still, doctors' perceptions may change as they learn to handle and prevent Lemtrada's side effects, said Eva Havrdova, a researcher at Charles University in Prague who presented safety data for the drug in Lyon.
"Neurologists may feel threatened, but they will have to understand how the immune system works and how to handle, so I don't think autoimmunity will be a big problem," she said.
Multiple sclerosis is a chronic, often disabling disease that attacks the central nervous system and can lead to numbness, paralysis and loss of vision. It affects 2.5 million people worldwide and has no cure.
But fresh data from existing treatments and the arrival new drugs is expected to transform the way doctors treat the disease. In the past, the severity of MS drugs' side effects have led 35 to 40 percent of sufferers to opt for no treatment at all.
"Now we are getting lots of therapies that do different things, have different benefits and risks, different means of administration and approaches to the disease," said Panzara. "This is a big time for change in this therapeutic space."
(Reporting by Elena Berton; Editing by Christian Plumb)