Duchenne Muscular Dystrophy market to shoot up 100-fold in five years

As a whole host of new Duchenne Muscular Dystrophy therapies mature down the regulatory pathway, the global market for the degenerative disease is expected to expand at an incredible rate – from $8.2 million in 2014 to $990 million in 2019, or so says market research firm GlobalData. The present market’s got only paltry offerings to treat this […]

As a whole host of new Duchenne Muscular Dystrophy therapies mature down the regulatory pathway, the global market for the degenerative disease is expected to expand at an incredible rate – from $8.2 million in 2014 to $990 million in 2019, or so says market research firm GlobalData.

The present market’s got only paltry offerings to treat this condition – hence the huge growth projection. And it could expand even further, because the early entrants into the DMD drug market only treat a small subset of patients – so more sophisticated therapies will be in demand, GlobalData said.

The immediate market uptick will occur largely because of the market entry of therapies from Sarepta Therapeutics, PTC Therapeutics and BioMarin, GlobalData said.

“These mutation-specific drugs are expected to have a high price point due to their novelty, efficacy and orphan drug status, and will contribute 85.6% to the DMD treatment arena by 2019,” GlobalData analyst Nikhilesh Sanyal said in a statement.

There are 23 companies around the world developing a DMD treatment, according to a count from the Timmerman Report.

Sarepta’s developing an RNA-based exon-skipping biologic that repairs the error or mutation in the dystrophin gene that causes the disease. Despite a newly ousted CEO and, for some, a dubious outlook for its therapy’s efficacy, the company’s midway through Phase 2 trials and still holds much promise to the DMD masses.

PTC’s drug, Translarna, also addresses the so-called “nonsense” mutation found in DMD – and this small molecule drug is thought to interact with the ribosome to allow it to pass over the faulty bits of gene and produce functional proteins. It’s received orphan status from the FDA and conditional marketing approval in the EU.

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BioMarin’s teamed up with the Netherlands-based Prosensa for another RNA-based DMD therapy that has made it through Phase 3, and is just awaiting approval in the U.S. and European approval. However, looks like it only is effective in a fragment of DMD patients – so, as the GlobalData report points out, there’s still a large market for drugs that will fill in the gaps. However, Sanyal says:

“There is currently a lack of standardization for measuring clinical efficacy across all stages of DMD. While most studies use the change in the six-minute walk test as the primary clinical endpoint, this applies only to ambulant patients and therefore excludes non-ambulant individuals, as well as children under the age of five. Furthermore, the high cost of novel disease-modifying drugs means that their reimbursement by local health authorities and insurance companies may be prevented following their entry into the arena.”