Genzyme welcomes new Health Canada drug rules on rare diseases

Biotech company Genzyme Canada is applauding a move by Ottawa to speed up approval and access to orphan drugs for patients with rare diseases, but new regulations will determine the size of any new investments at its growing Mississauga plant.

The Canadian unit of French drug giant Sanofi-Aventis did not specify whether the Health Canada announcement earlier Wednesday means more money for research or potential new markets for the pharmaceutical industry.

But general manager Peter Brenders told it would now be easier for Genzyme and other early-stage biotech companies to bring products to Mississauga residents and other Canadians.

“We’re going to be able to support more research in the field,” Brenders, 47, said in an interview from Genzyme’s head office on Matheson Boulevard East.

He also said Genzyme Canada is already expanding its Mississauga headquarters and lab, which employ about 50 people.

“As we get ready to prepare to launch some products for multiple sclerosis and stuff we see potentially coming down the pipeline (that) will allow us to see where we need to grow as well.”

An orphan drug is a medicine developed specifically to treat rare diseases such as Fabry, Gaucher, Pompe and MPS. About 2.8 million Canadians are believed to suffer from such disorders.

In Ottawa, Health Minister Leona Aglukkaq announced the federal government will create a new approach for the authorization of orphan drugs and began the Canadian launch of Orphanet — a comprehensive database of information and services for rare diseases.

“Our government will introduce a new approach that will better support the development and authorization of drugs for rare diseases and launch of a new Web portal to assist patients in finding the information and services they need,” the minister said.

“The new framework will be designed to spur research and innovation in Canada. Building on existing international experience and knowledge, it will provide Health Canada with new tools to gather and share information, including the registration of clinical trials.”

Today, when an orphan drug is unavailable in Canada, doctors can apply individually for each patient through Health Canada’s special access program.

At Genzyme, Brenders is also pleased the industry will now be able to access the Orphanet site to find researchers drug companies can work with and what development programs are out there that Genzyme can join.

“It’s a way that makes it easier for companies to bring products to patients,” he said.

“Development of products for rare diseases, like many products, is very time consuming, very expensive, very arduous in terms of the workload involved. Many products take a lot of time to work. The more that we know, the more that we connect, the faster it’s going to be. We’re expediting care.”

Brenders, who joined Genzyme five months ago after serving as president and CEO of national industrial biotechnology association BIOTECanada, said having special recognition for these diseases, as there is in the U.S.  and Europe, is important in helping recognize the need to have a slightly different process so Canadian patients can get quicker access to new therapies.

“When the regulations come out and we see there’s a defined process for rare disease treatments that just means that companies like Genzyme, that want to bring it to Canadians, have a nice clear process that opens the door for these treatments,” he said.

Genzyme was a former U.S. company acquired by French multinational Sanofi in 2011 for $20.1 billion. Genzyme employs more than 11,000 people around the world, 50 of them in Mississauga.

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