The FDA has awarded orphan drug designation to Cure Rare Disease’s CRD-003 for the treatment of limb-girdle muscular dystrophy type R9 (LGMD2i/R9), a congenital muscular dystrophy caused by biallelic mutations in the FKRP gene.
Sonomind SAS raised €3 million (US$ 3.5 million) in financing for its transcranial neuromodulation system, which targets deep regions within the brain non-invasively. The funds will go towards accelerating the development of the platform, which has already proven to be safe and effective in treating patients suffering from drug-resistant depression, after only five days of treatment.
Uniqure NV rang the bell with a best-case scenario in the pivotal phase I/II study with AMT-130 for the treatment of Huntington’s disease, and shares of the Amsterdam-based firm (NASDAQ:QURE) closed Sept. 24 at $47.50, up $33.84, or 248%. The study met its prespecified primary endpoint, with high-dose AMT-130 turning up a statistically significant slowing of disease progression as measured by the composite Unified Huntington’s Disease Rating Scale at 36 months compared to a propensity score-matched external control.
Amyotrophic lateral sclerosis (ALS), formerly known as Lou Gehrig’s disease, is a progressive and fatal neurodegenerative disorder with no known cure. While three therapies have gained U.S. FDA approvals to date, including Rilutek (riluzole), Radicava/Radicava ORS (edaravone) and tofersen (BIIB-067, the lack of a disease-modifying drug has spurred the continual search for novel therapies.
The first new U.S. FDA-approved therapeutic option for PTSD in more than 20 years will have to wait. A supplemental NDA seeking approval of Rexulti (brexpiprazole) combined with sertraline, filed by one of Japan’s biggest pharmas, Otsuka Pharmaceutical Co. Ltd., received a complete response letter (CRL) from the agency.
Breaking with its long-held, oft-recited mantra that observational studies are great for generating hypotheses but not as evidence for approval, the U.S. FDA is initiating the approval of leucovorin calcium tablets for patients with cerebral folate deficiency, a neurological condition that affects folate transfer into the brain.
Experts agree that the earlier Alzheimer's disease is detected, the sooner action can be taken. And so, the key to preventing deterioration is identifying the most effective early biomarkers, those that can spot the disorder and help halt its progression. Recent advances in the field have pushed a new era of early detection through blood-based biomarkers and personalized medicine strategies based on each patient’s genetic, immunological and clinical profile.
The Royal College of Psychiatrists in the U.K. has published its first-ever guidance to support research into psychedelic drugs as therapies for conditions including treatment-resistant depression, substance abuse disorders and posttraumatic stress disorder, saying that in a fast-moving field there is a risk of jumping ahead of the evidence.
Humanwell Healthcare (Group) Co. Ltd. has divulged P2X purinoceptor 7 (P2RX7; P2X7) antagonists reported to be useful for the treatment of pain, inflammatory, neurological and immunological disorders.
As the first – and so far only – drug to enter clinical testing for the rare neurogenetic disorder Alexander disease, there were some unknowns heading into the readout of the pivotal study testing Ionis Pharmaceuticals Inc.’s zilganersen in children and adults. But the top-line data yielded a clear win for the antisense oligonucleotide candidate, which demonstrated a disease-modifying impact, including statistical significance on the primary endpoint of gait speed as assessed by the 10-Meter Walk Test vs. control at week 61.
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