Aug 4 PTC Therapeutics Inc said
European regulators have given conditional approval to its
muscle wasting disorder drug, sending its shares up 4.5 percent
in early trading on Monday.
The approval is the first in the world for a drug designed
to treat the underlying cause of Duchenne muscular dystrophy
(DMD) - a genetic disorder that causes muscle degeneration and
eventual death in boys.
Conditional marketing authorization of a drug allows for the
marketing of medicines designed to treat life-threatening
diseases that have few or no treatment options, even if
comprehensive clinical data is not yet available.
As part of the conditional approval, PTC is obliged to
complete a confirmatory late-stage trial of the drug,
Translarna, and submit additional efficacy and safety data.
Translarna is designed to interact with a part of the cell
known as a ribosome, enabling it to manufacture a protein called
dystrophin, the absence of which causes muscle weakening in
patients with DMD.
About 15 percent of DMD cases are caused by "nonsense
mutations" that lead to a premature end to the translation of a
gene into a protein, according to the U.S. National Institutes
The approval is for ambulatory patients aged five years and
PTC shares were up 3.4 percent at $28.74 in mid-morning
trading on the Nasdaq.
(Reporting by Natalie Grover in Bangalore)