After Sarepta Fate, Another Disappointment for Duchenne Patients


FDA decision on Sarepta Therapeutics’ eteplirsen was up in the air and yet another bad news came for the Duchenne Muscular Dystrophy (DMD) patients.

biomarin pharmaceutical logoBioMarin Pharmaceutical have withdrawn the Marketing Authorization Application (MAA) from the European Medicines Agency (EMA) for its investigational Duchenne drug, Kyndrisa™ (drisapersen) intended for the treatment of DMD.

In January, Kyndrisa received Complete Response Letter from US FDA and later the discussions at the Committee for Medicinal Products for Human Use (CHMP) meeting held in May “clearly indicated that CHMP intended to issue a negative opinion”. These factors compelled BioMarin to wrap up Kyndrisa.

The withdrawal of the MAA and discontinuation of our current experimental drugs for Duchenne is a difficult but necessary decision at this time,” said Jean-Jacques Bienaimé, BioMarin chairman and chief executive officer. “We want to extend our sincere gratitude to all of the families and caregivers who supported our efforts over the last year to bring Kyndrisa to patients with Duchenne. Our plan now is to invest in research of next generation oligonucleotides with the goal of making a safe and effective treatment available for boys with this devastating disorder“.

BioMarin is a global biotechnology company dedicated towards development and commercialization of innovative therapies for the life-threatening rare disorders. Based on the similar factors responsible for the doom of Kyndrisa, BioMarin also intends to wrap up its three investigational candidates (BMN 044, BMN 045 and BMN 053) in its pipeline for the treatment of DMD.  All of them have received orphan drug status in EU and US and are currently in their clinical development phases.

Biomarin intends to figure out a plan for the transition of patients currently being treated with Kyndrisa, BMN 044, BMN 045 and BMN 053.

According to a statement released, “The Company will continue to explore the development of next generation oligonucleotides for the treatment of Duchenne muscular dystrophy”.

Drisapersen became a part of BioMarin’s portfolio in 2014 when they acquired Prosensa Holding N.V. in $680 million deal. It had already received Orphan, Fast Track and Breakthrough designation at that time. The major driving force behind the doom of Kyndrisa was the renal abnormalities including subclinical proteinuria occurred during the clinical trial programme.

DMD is a genetic, fatal disorder occurs to approximately 1 in 3,500 newborn boys. It leads to progressive muscle degradation due to the absence of a protein, dystrophin which is required to keep the muscle cells intact.


Figure 1. Cause of DMD (Photo credit: “Designer” genes correct Muscular Dystrophy, //

Kyndrisa is a RNA-based product to induce exon 51 skipping in dystrophin gene which restores the proper dystrophin reading frame in DMD patients.
BioMarin’s Kyndrisa was in competition with Sarepta’s Duchenne drug eteplirsen. Eteplirsen also have few safety concerns; patients reported increased level of the muscle strengthening protein, dystrophin. The FDA decision on eteplirsen is also pending but if approved, the doom of Kyndrisa could increase the commercial opportunity of eteplirsen.

Featured image credit: Desperate man on night city blurred lights © zurijeta (Stock Photo ID: 82493408)

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