NEW YORK – Roche this week said that the European Medicines Agency has begun reviewing a marketing authorization application for adeno-associated virus therapy Elevidys (delandistrogene moxeparvovec) as a treatment for patients 3 to 7 years old with Duchenne muscular dystrophy. Roche inked a deal in 2019 with Sarepta Therapeutics to seek regulatory approval and market the drug outside the US. The application is based on results from the Phase III EMBARK trial, which demonstrated a clinically meaningful and statistically significant improvement in time to rise from the floor, 10-minute walk test, and stride velocity. Last week, the US Food and Drug Administration converted its earlier accelerated approval of Elevidys to full approval and expanded the indication to cover older patients.
Rockville, Maryland-based Regenxbio this week said it has expanded its Phase I/II AFFINITY DUCHENNE clinical trial, in which it is testing its experimental Duchenne muscular dystrophy gene therapy RGX-202 and has begun enrolling a new cohort of younger patients ages 1 to 3 years old. The company expects data from this cohort to be part of its pivotal program plans and biologics license application, in which it will seek a broad label. Regenxbio has scheduled an end-of-Phase II meeting with the US Food and Drug Administration for late July to finalize the design of a pivotal trial, which remains on track to launch in the second half of the year.
AstraZeneca this week said that Tagrisso (osimertinib) has been approved in China and Japan as a first-line treatment for patients with locally advanced or metastatic EGFR-mutated non-small cell lung cancer with exon 19 deletions or exon 21 (L858R) mutations. The regulatory agencies based their decisions on results from the FLAURA2 Phase III trial, in which Tagrisso plus chemotherapy reduced the risk of disease progression or death by 38 percent compared to Tagrisso alone.
New York City-based BrainStorm Cell Therapeutics this week said it has reached alignment with the US Food and Drug Administration on the chemistry, manufacturing, and controls (CMC) aspects of its Phase IIIb clinical trial evaluating its investigational autologous stem cell therapy NurOwn (debamestrocel) in amyotrophic lateral sclerosis (ALS), which the company expects to use to support a biologics license application (BLA). BrainStorm has faced multiple setbacks in its effort to win regulatory approval for NurOwn, including withdrawing a BLA with the FDA late last year on the heels of negative feedback from the agency and an advisory committee of independent experts.
The US Food and Drug Administration this week granted fast-track designation to Carisma Therapeutics' ex vivo gene-modified autologous cell therapy, CT-0525, as treatment for HER2-overexpressing solid tumors. Carisma is evaluating the treatment, a CAR-monocyte therapy, in a Phase I trial enrolling previously treated patients with HER2-overexpressing disease. A sponsor can meet with the FDA more frequently to get advice on developing fast-track designated agents, submit data on a rolling basis, and apply for accelerated approval or priority review.
Turkish genomics healthcare company Genoks and precision medicine-focused informatics firm Velsera said they are collaborating to advance cancer diagnostics and treatment in Turkey and nearby regions. The partnership combines Genoks' genomic expertise with Velsera's Clinical Genomics Workspace (CGW), a platform designed for comprehensive analysis of data obtained from solid tissue and liquid tumor as well as hematological cancer samples. Genoks will integrate CGW into its cancer diagnostic services, enhancing its ability to detect and characterize various types of cancer accurately, the companies said.
The US Food and Drug Administration has granted ReCode Therapeutics orphan drug designation for its investigational candidate RCT1100, which it is developing as a treatment for primary ciliary dyskinesia (PCD) caused by certain mutations in the DNAI1 gene, the Menlo Park, California-based company said this week. RCT1100 is an mRNA therapy that's inhaled and designed to deliver DNAI1 mRNA to airway cells. ReCode is currently testing the candidate in an open-label Phase I clinical trial to assess safety and tolerability. Mutations in DNAI1 account for about 7 percent of cases of PCD, a rare and progressive genetic lung disorder.
In Brief This Week is a selection of news items that may be of interest to our readers but had not previously appeared on Precision Medicine Online.