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Cure Sanfilippo agrees: The need to use surrogate biomarkers in an accelerated drug approval pathway for diseases like Sanfilippo

July 8, 2021

A powerful article from Dr. Emil D. Kakkis outlines the critical need for use of surrogate biomarkers in an accelerated drug approval pathway for diseases like Sanfilippo syndrome.

In the article, “Aduhelm’s accelerated approval offers a promising roadmap for rare neurological diseases,” Dr. Kakkis, a distinguished scientist, industry leader, and rare disease advocate, illustrates how the use of biomarkers revolutionized and sped the approval of treatments for HIV and cancer patients.

“When it comes to making new therapies for complex, difficult-to-treat diseases, history has shown that progress can’t be made without taking a first — often controversial — step,” states Dr. Kakkis in the article. “Without the FDA’s accelerated approval program and novel first treatments based on new and imperfect biomarker endpoints, HIV would not be a controllable disease today, and we might not have such a flourishing clinical research ecosystem in oncology.”

“Aduhelm’s accelerated approval offers a promising roadmap for rare neurological diseases,” Dr. Emil Kakkis

Cure Sanfilippo Foundation and the patient community believe that it is imperative that children with Sanfilippo syndrome and other degenerative rare diseases are provided this same urgent consideration; not because we expect that one drug approval will resolve every need for our children who are suffering, but because it will be a bridge and will spur more innovation and advancement in diagnostics and therapeutics; that we might hope for better and better treatments as science progresses.

“We can’t continue to wait to take this first step, particularly when the need is so urgent and the disease so devastating,” said Dr. Cara O’Neill, Chief Science Officer for and co-founder of Cure Sanfilippo Foundation.

Accelerated drug approval pathways using relevant disease biomarkers allow treatments with potential benefit to be available to patients whose disease process does not allow them to wait for full approval, while longer term studies are carried out to define the full benefit.

“If use of the heparan sulfate level in cerebrospinal fluid was to be recognized as a biomarker that reflects the underlying disease activity, it could accelerate a number of therapeutic programs [for Sanfilippo syndrome] that are now at risk for never being approved,” states Dr. Kakkis in the article.

“We encourage the FDA to use the flexibility in their hands to accelerate therapies getting to those in need and applaud the examples where this flexibility was used,” said Glenn O’Neill, president and co-founder of Cure Sanfilippo Foundation.

Read the article by Dr. Kakkis.

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