This is an article about Dr. Kakkis who is the CEO of Ultragenyx, the bio-pharma company that is conducting the clinical trial for HIBM with Sialic Acid. There is hope that this medicine will stop the progression and help re-energized the muscles.
p.s. please check my previous post and donate if you can to the cause I doing a fund raising. Your donation is tax deductible and will help the ARM/HIBM organisation to further gene therapy for HIBM.
Emil Kakkis is making another run at rare diseases.
The president and CEO of Ultragenyx Pharmaceutical Inc. and former chief medical officer at BioMarin Pharmaceutical Inc. is laying out a new strategy Tuesday to get more drugs into children with rare diseases. Under the banner of the EveryLife Foundation for Rare Diseases, which Kakkis founded and has largely bankrolled, he is proposing that companies with already-approved, big-market drugs be rewarded with a two-month extension of that drug’s patent life if it wins a new Food and Drug Administration-approved label in an orphan disease.
“What if every, single blockbuster cancer drug had three or four pediatric indications in development to gain patent extensions for the whole franchise?” Kakkis wrote in an email.
It isn’t just random wondering for Kakkis, who at UCLA co-discovered a treatment for the lysosomal storage disease MPS and followed the drug to San Rafael-based BioMarin (NASDAQ: BMRN). He left BioMarin a dozen years later to start Ultragenyx, which has raised more than $120 million over the past two years and will complete three Phase II studies in rare diseases this year.
Kakkis’ plan makes sense for drug makers, he said, because those companies already have expertise in the drugs, can move development along quickly and can be rewarded with an important extra few months of no-competition sales of the drug for its main use.
“It’s so easy for them to do, and the value is so significant,” Kakkis said in an interview. “It’s an efficient way to generate real improvement. There’s no new manufacturing process or toxicology process or trying to figure out the indication.”
For patients, Kakkis estimates that research into hundreds of patented drugs will be reopened for uses in rare diseases. What’s more, the drugs likely would cost less than the $200,000-and-up price of many of today’s start-from-scratch rare disease treatments, because much of the safety and toxicology work will have already been done.
And for Kakkis’ Novato company? Nothing.
“I’m trying to do the right things,” Kakkis said. “It doesn’t solve all rare diseases. It solves some of them that can use a common drug.”
It’s no accident that Kakkis is laying out his plan around Rare Disease Day, which is Thursday, and this week's Rare Disease Legislative Advocates conference in Washington, D.C. Some 150 advocates are expected to visit their elected leaders on Capitol Hill, compared to 70 last year.
“The rare disease lobby is getting stronger and more vocal,” Kakkis said.
Indeed, patients, caregivers, doctors and researchers made their voices heard in FDASIA, last year’s legislation that laid out the fee structure for seeking approval of their drugs. The legislation established a new Office of Drug Evaluation, specifically for biochemical and genetic diseases, and created new criteria that could accelerate approvals for rare disease treatments.
Still, it’s not enough, said Kakkis, who recently committed $400,000 of his own money to the EveryLife Foundation.
Kakkis is laying out his plan as a 2.0 version of the “Cure the Process” campaign that was launched in spring 2009. Along with patent extensions, he wants to “rationalize” safety testing, aligning U.S. guidelines with those in Europe. The EveryLife Foundation also is writing up its version of accelerated approval guidelines that it hopes the FDA will adopt.
“The people who do the work win,” Kakkis told supporters at a fundraiser before last month’s J.P. Morgan Healthcare Conference. “Patients need it. We all need it.”
p.s. please check my previous post and donate if you can to the cause I doing a fund raising. Your donation is tax deductible and will help the ARM/HIBM organisation to further gene therapy for HIBM.
Man with a plan: Emil Kakkis aims to lure big-market drugs toward rare diseases
- Ron Leuty
- Reporter- San Francisco Business Times
- Email | Biotech tweets | Google+ | Education tweets
The president and CEO of Ultragenyx Pharmaceutical Inc. and former chief medical officer at BioMarin Pharmaceutical Inc. is laying out a new strategy Tuesday to get more drugs into children with rare diseases. Under the banner of the EveryLife Foundation for Rare Diseases, which Kakkis founded and has largely bankrolled, he is proposing that companies with already-approved, big-market drugs be rewarded with a two-month extension of that drug’s patent life if it wins a new Food and Drug Administration-approved label in an orphan disease.
“What if every, single blockbuster cancer drug had three or four pediatric indications in development to gain patent extensions for the whole franchise?” Kakkis wrote in an email.
It isn’t just random wondering for Kakkis, who at UCLA co-discovered a treatment for the lysosomal storage disease MPS and followed the drug to San Rafael-based BioMarin (NASDAQ: BMRN). He left BioMarin a dozen years later to start Ultragenyx, which has raised more than $120 million over the past two years and will complete three Phase II studies in rare diseases this year.
Kakkis’ plan makes sense for drug makers, he said, because those companies already have expertise in the drugs, can move development along quickly and can be rewarded with an important extra few months of no-competition sales of the drug for its main use.
“It’s so easy for them to do, and the value is so significant,” Kakkis said in an interview. “It’s an efficient way to generate real improvement. There’s no new manufacturing process or toxicology process or trying to figure out the indication.”
For patients, Kakkis estimates that research into hundreds of patented drugs will be reopened for uses in rare diseases. What’s more, the drugs likely would cost less than the $200,000-and-up price of many of today’s start-from-scratch rare disease treatments, because much of the safety and toxicology work will have already been done.
And for Kakkis’ Novato company? Nothing.
“I’m trying to do the right things,” Kakkis said. “It doesn’t solve all rare diseases. It solves some of them that can use a common drug.”
It’s no accident that Kakkis is laying out his plan around Rare Disease Day, which is Thursday, and this week's Rare Disease Legislative Advocates conference in Washington, D.C. Some 150 advocates are expected to visit their elected leaders on Capitol Hill, compared to 70 last year.
“The rare disease lobby is getting stronger and more vocal,” Kakkis said.
Indeed, patients, caregivers, doctors and researchers made their voices heard in FDASIA, last year’s legislation that laid out the fee structure for seeking approval of their drugs. The legislation established a new Office of Drug Evaluation, specifically for biochemical and genetic diseases, and created new criteria that could accelerate approvals for rare disease treatments.
Still, it’s not enough, said Kakkis, who recently committed $400,000 of his own money to the EveryLife Foundation.
Kakkis is laying out his plan as a 2.0 version of the “Cure the Process” campaign that was launched in spring 2009. Along with patent extensions, he wants to “rationalize” safety testing, aligning U.S. guidelines with those in Europe. The EveryLife Foundation also is writing up its version of accelerated approval guidelines that it hopes the FDA will adopt.
“The people who do the work win,” Kakkis told supporters at a fundraiser before last month’s J.P. Morgan Healthcare Conference. “Patients need it. We all need it.”
Ron Leuty covers biotech, higher education and China for the San Francisco Business Times.
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