RDR Alert®

The U.S. FDA has accepted for priority review the Biologics License Application for cemiplimab (REGN2810), an investigational anti-PD-1 monoclonal antibody for patients with metastatic CSCC.

This morning, Capricor Therapeutics, Inc. announced that the HOPE-2 clinical trial has been initiated at UC Davis Medical Center in Sacramento, CA.

Treatment with volanesorsen shows efficacy in patients with FCS.

Results from a questionnaire show that individuals living with FCS prove that communication among people in similar situations can improve a patient’s quality of life.

At the NLA Scientific Sessions, Akcea Therapeutics presented a poster with data from 2 different studies, each of which exhibited a significant reduction of triglycerides in patients with FCS when administered volanesorsen.

At the NLA Scientific Sessions in Las Vegas this morning, Akcea Therapeutics, presented a poster with data from the largest burden of illness study in patients with FCS to date.

At the NLA Scientific Sessions in Las Vegas, NV this weekend, Akcea Therapeutics will present clinical data on volanesorsen and FCS.

This morning, biotechnological company Saniona announced that it has obtained approval to initiate the second part of its Phase 2a trial for Tesomet in adolescents with Prader-Willi syndrome (PWS).

This morning, top-line data from a confirmatory Phase 2 study of NP001 in ALS were reported, and showed that the trial did not meet its primary or secondary endpoints.

Dystrophic epidermolysis bullosa treatment receives IND clearance.

Data from Type 1 spinal muscular atrophy (SMA) treatment suggests efficacy and tolerability.

CLN2 disease treatment reduces rate of clinical decline in children.

IONIS-HTT data shows link between reduction in Huntingtin protein and clinical improvement.

FLT3mut+ Relapsed/Refractory AML treatment submits New Drug Application.

Potential PKU treatment receives FDA Fast Track designation.

This morning, Lin BioScience announced that the US FDA has granted orphan drug designation to LBS-007 for the treatment of acute lymphoblastic leukemia (ALL).

Progeria treatment extends survival in children.

Nusinersen treatment proven to be efficient in spinal muscular atrophy (SMA).

An observational study in Dutch CAPS patients was conducted by Catharina M. Mulders-Manders and colleagues, and results were published in the Orphanet Journal for Rare Diseases.

At the 70th Annual AAN 2018 Annual Meeting, Alnylam announced new results from the APOLLO Phase 3 study of patisiran, an investigational RNAi therapeutic for the treatment of hATTR amyloidosis.

AbobotulinumtoxinA proves effective across multiple therapeutic uses.

New data proves efficacy of relapsing multiple sclerosis (RMS) treatment.

The U.S. FDA has cleared the Investigational New Drug application for DTX401 for the treatment of glycogen storage disease type Ia (GSDIa).

Final approval was given this morning to an Abbreviated New Drug Application for Miglustat 100 mg capsules for the treatment of adult patients with mild to moderate type 1 Gaucher disease.

ABO-102, which has previously received rare pediatric disease designation, fast track designation, and orphan drug designation from the U.S. FDA, was the recipient of another acknowledgement this morning when it was granted RMAT designation.

Dr Ali Fatemi, MD, of Kennedy Krieger Institute and John Hopkins University, describes the “Awesome Disease” (LBSL) and how a young girl now inspires his research.

At the 70th AAN Annual Meeting this morning Summit Therapeutics plc presented new 24-week interim data from PhaseOut DMD, its Phase 2 trial of ezutromid in Duchenne.

The FDA approved fostamatinib disodium hexahydrate (Tavalisse) tablets for the treatment of thrombocytopenia in adult patients with chronic immune thrombocytopenia. RDR discusses with Dr James Bussel.

Researchers from GlaxoSmithKline will collect data from a group of young children treated with Strimvelis in Milan.

On Wednesday, it was announced that Synlogic had initiated and dosed the first subject in its Phase 1/2a study of SYNB1618 for the treatment of phenylketonuria.