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Physicians can suggest Risdiplam for spinal muscular
atrophy (SMA) treatment in patients aged two months and older.
Recently, the first oral drug- Risdiplam was approved by the FDA on 7 August 2020, for the treatment of spinal muscular atrophy (SMA) patients aged two months and older. SMA is a rare and common fatal genetic disease affecting muscle strength and movement.
Risdiplam encompasses a survival of motor neuron 2-directed
RNA splicing modifier. Two clinical trials have investigated its efficacy in
patients suffering infantile-onset and later-onset SMA. Twenty-one patients
(mean age of 6.7 months) were included in infantile-onset SMA study. Efficacy
was proved as per the ability to sit without support for at least 5 seconds and
survival without lasting ventilation in this open-label study. As found, 41% of
patients were able to sit individually for >5 seconds after a year of
treatment. Also, 81% of patients were alive without lasting ventilation, which
is an obvious improvement from characteristic disease progression without
treatment after 23 or more months.
In the second randomized, placebo-controlled study, a
total of 180 patients with SMA aged 2 to 25 years were included. The change
from starting in MFM32 (a test of motor function) total score at the 1-year
mark was regarded as the primary endpoint. The patients using Risdiplam had a
1.36 rise on an average in their score at the 1-year mark than a 0.19 decrease
in patients on placebo (inactive treatment).
Rash, fever, diarrhoea, ulcers of the mouth area, joint
pain and urinary tract infections (UTI) were frequently observed side effects
of Risdiplam. Similar side effects were observed in cases of infantile-onset
SMA and later-onset SMA. Additionally, UTI, pneumonia, vomiting and
constipation were prevalent in infantile-onset.
As risdiplam may increase plasma concentrations of some
drugs, therefore, patients should avoid taking risdiplam together with drugs
that are multidrug and toxin extrusion substrates.
Risdiplam obtained orphan drug designation, which offers
reasons to promote and boost drug development for rare disorders. The
application was presented a Rare Pediatric Disease Priority Review Voucher.
FDA
FDA Approves Oral Treatment for Spinal Muscular Atrophy
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