by Angela Maas
Since 2016, the FDA has approved a handful of therapies to treat
Duchenne muscular dystrophy (DMD). But some uncertainty exists over their
effectiveness, in addition to concerns about their costs.
When DMD is suspected, a blood test that measures creatine
kinase (CK) levels is performed. "CK is an enzyme found in abnormally high
levels when muscle is damaged," says Mesfin Tegenu, R.Ph., president of
PerformRx, LLC. "The detection of an elevated CK level leads to molecular
genetic testing to confirm a definitive diagnosis of DMD."
In February 2017, the FDA approved then-manufacturer Marathon
Pharmaceuticals LLC's Emflaza (deflazacort). The company said it would be
priced at $89,000, which sparked outrage since people have been buying a
generic version from overseas since the 1990s for about $1,000 per year. After
the backlash, Marathon ultimately sold the drug to PTC Therapeutics Inc., which
launched it later that year with a $35,000 annual price tag. Since then, PTC
has raised the price to more than Marathon’s original one.
In September 2016, the FDA gave accelerated approval to Sarepta
Therapeutics, Inc.'s Exondys 51 (eteplirsen). The dystrophin gene has 79 exons,
and about 80% of people with DMD have genotypes that are amenable to exon
skipping. Exondys 51 targets those with a mutation of the DMD gene that is
amenable to exon 51 skipping.
Sarepta also has a second exon-skipping therapy, Vyondys 53
(golodirsen), which treats DMD in people with a mutation amenable to exon 53
skipping. The FDA gave the drug accelerated approval in December, almost four
months after the agency rejected the drug through a complete response letter.
Both drugs are weight-based with similar prices: about $300,000
per year but up to $1 million annually.
"It's unclear how much a health plan may spend on someone
with DMD; however, a recent study from the Muscular Dystrophy Association found
that the annual cost for DMD for U.S. society as a whole is around $362-$488
million dollars," says Tegenu. "The price of the newer DMD therapies
(Exondys 51 and Vyondys 53) are both estimated to cost approximately $750,000
per year for the treatment of one patient."
Pharma manufacturers continue to focus on the DMD space, and
several products, including gene therapies, are in the pipeline.
From
RADAR on Specialty Pharmacy
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