Europe gives green light to 1st gene therapy for children

FDA proposes limit on inorganic arsenic in infant rice cereal

LONDON, April 2, (RTRS): The world’s first life-saving gene therapy for children, developed by Italian scientists and GlaxoSmithKline, has been recommended for approval in Europe, boosting the pioneering technology to fix faulty genes.

The European Medicines Agency (EMA) said on Friday it had endorsed the therapy, called Strimvelis, for a tiny number of children 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 the ultra-rare genetic disorder, which leaves them unable to make a type of white blood cell. They rarely survive beyond two years unless their immune function is restored with a suitable bone marrow transplant.

SCID is sometimes known as “bubble baby” disease, since children born with it have immune systems so weak they must live in germ-free environments.

Strimvelis is expected to secure for- mal marketing authorisation from the European Commission in a couple of months, making it the second gene therapy to be approved in Europe, after UniQure’s Glybera, which treats a rare adult blood disorder.

The US Food and Drug Administration has yet to approve any gene therapies but a growing number of US biotech companies, such as Bluebird Bio, have products in development.

Other large pharmaceutical companies are also eyeing the field, including Bristol-Myers Squibb, which has a tieup with UniQure.

Research into gene therapy goes back a quarter of a century but the field has experienced many setbacks, including the high-profile death of an American patient in 1999 and some disastrous clinical trial results in the late 1990s and early 2000s.

Now, though, optimism is building, helped by the discovery of better ways to carry replacement genes into cells.

Martin Andrews, head of GlaxoSmithKline’s rare diseases unit, believes the technology is proving itself, although it remains at an early stage of development.

“We’re on page one of chapter one of a new medicine text book”, he told Reuters.

A host of challenges still need to be overcome, including the 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 a therapy like Strimvelis. UniQure’s Glybera made history in 2014 as the first drug to carry a $1 million price tag. GSK is not putting a price on its product but a source close to the company said that, if approved, Strimvelis would cost “very significantly less than $1 million”.

GSK has several other gene therapies under development with researchers at Fondazione Telethon and Ospedale San Raffaele in Italy, including treatments for metachromatic leukodystrophy and Wiskott-Aldrich syn- drome that could be submitted for regulatory approval in the next couple of years.

Its Strimvelis treatment for ADASCID is also being lined up for submission to US regulators, although Andrews said this would not happen before the end of next year.

WASHINGTON:

The US Food and Drug Administration said on Friday it was taking steps to cut inorganic arsenic levels in infant rice cereal, a primary source of arsenic exposure in infants.

Exposure to inorganic arsenic in infants and pregnant women can result in decreased performance by children on developmental tests that measure learning.

The FDA is proposing a limit of 100 parts per billion (ppb) in the cereal, in line with the level set by the European Commission for rice intended for infants and young children.

The agency said its tests found that most cereals on the market either meet or are close to the proposed level.

The FDA said it was not recommending that the general population change their current rice consumption patterns, but was offering targeted information for pregnant women and infants to help reduce exposure.

Infant rice cereal products made by Nestle SA’s unit Gerber already meet the limit proposed by the FDA, Wendy Johnson-Askew, a spokeswoman for the food products giant, said in an email.

Other manufacturers including Abbott Laboratories and Kraft Heinz Co did not immediately respond to requests for comment.

Influential consumer products magazine Consumer Reports urged the

in 2012 to set limits for arsenic in rice after tests on more than 60 popular products showed that most contained inorganic arsenic.

Consumer Reports said on Friday it welcomed the proposed limit, but remained “concerned that so many other rice-based products consumed by children and adults remain without any standards at all”.

The magazine will continue to push the FDA to set levels for these products, especially ready-to-eat cereal for children, it said.

Arsenic exists in two forms, naturally occurring organic and inorganic, which is often used in feed for poultry and occasionally hogs to prevent disease.

Waste from those animals can contaminate fields and waterways when it is used as fertilizer. As a result, arsenic can be found in rice, fruit, vegetables and seafood — all of which are considered healthy.

Exposure to inorganic arsenic in rice and rice products causes an additional four cases of lung and bladder cancer over the lifetime of every 100,000 people in the United States, the FDA said.

These cases would account for far less than 1 percent of the nation’s total, the agency said.

The proposed limit is open for public comment for 90 days.