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By Ben Hirschler
LONDON, April 1 (Reuters) - The world's first life-savinggene therapy for children, developed by Italian scientists andGlaxoSmithKline, has been recommended for approval inEurope, boosting the pioneering technology to fix faulty genes.
The European Medicines Agency (EMA) said on Friday it hadendorsed the therapy, called Strimvelis, for a tiny number ofchildren with ADA Severe Combined Immune Deficiency (ADA-SCID)for whom no matching bone marrow donor is available.
Around 15 children a year are born in Europe with theultra-rare genetic disorder, which leaves them unable to make atype of white blood cell. They rarely survive beyond two yearsunless their immune function is restored with a suitable bonemarrow transplant.
SCID is sometimes known as "bubble baby" disease, sincechildren born with it have immune systems so weak they must livein germ-free environments.
Strimvelis is expected to secure formal marketingauthorisation from the European Commission in a couple ofmonths, making it the second gene therapy to be approved inEurope, after UniQure's Glybera, which treats a rareadult blood disorder.
The U.S. Food and Drug Administration has yet to approve anygene therapies but a growing number of U.S. biotech companies,such as Bluebird Bio, have products in development.
Other large pharmaceutical companies are also eyeing thefield, including Bristol-Myers Squibb, which has atie-up with UniQure.
MANY SETBACKS
Research into gene therapy goes back a quarter of a centurybut the field has experienced many setbacks, including thehigh-profile death of an American patient in 1999 and somedisastrous clinical trial results in the late 1990s and early2000s.
Now, though, optimism is building, helped by the discoveryof better ways to carry replacement genes into cells.
Martin Andrews, head of GlaxoSmithKline's rare diseasesunit, believes the technology is proving itself, although itremains at an early stage of development.
"We're on page one of chapter one of a new medicine textbook," he told Reuters.
A host of challenges still need to be overcome, includingthe complexity of delivering a product like GSK's new treatment,which requires bone marrow cells to be taken from the patient,processed and injected back.
Trickiest of all may be pricing, given the tiny market for atherapy like Strimvelis. UniQure's Glybera made history in 2014as the first drug to carry a $1 million price tag. GSK is notputting a price on its product but a source close to the companysaid that, if approved, Strimvelis would cost "verysignificantly less than $1 million".
GSK has several other gene therapies under development withresearchers at Fondazione Telethon and Ospedale San Raffaele inItaly, including treatments for metachromatic leukodystrophy andWiskott-Aldrich syndrome that could be submitted for regulatoryapproval in the next couple of years.
Its Strimvelis treatment for ADA-SCID is also being lined upfor submission to U.S. regulators, although Andrews said thiswould not happen before the end of next year. (Writing by Ben Hirschler and Martinne Geller; Editing by DavidHolmes)
