dm+d
Unassigned
New Medicines
Nexviadyme (EU), Nexviazyme (US)
Late-onset glycogen storage disease type II (Pompe disease) in children and adultsInformation
Nexviadyme (EU), Nexviazyme (US)
New formulation
Sanofi
Sanofi
Development and Regulatory status
Phase III Clinical Trials
Approved (Licensed)
Approved (Licensed)
Yes
Jun 22
Approved in the EU [22].
Mar 22
MHRA renews its EAMS scientific opinion for avalglucosidase alfa in the treatment of late- and infantile-onset Pompe disease for patients who have already received enzyme replacement therapy with alglucosidase alfa, for a further 12 months [21].
Nov 21
Following a re-examination of its original opinion, the CHMP confirmed that avalglucosidase alfa is not considered a new active substance [19].
Aug 21
Sanofi aims to make Nexviazyme available in the US “in the coming weeks” at the same price as Lumizyme (avalglucosidase alfa-ngpt), which has a list price of about $905 per 50-mg injection [18].
Aug 21
Approved in US [16]
Jul 21
Sanofi requests a re-examination of EMA July 2021 opinion in relation to the conclusion of the CHMP that avalglucosidase alfa does not qualify as a New Active Substance [17].
Jul 21
EMA CHMP recommends use of Nexviadyme for long-term enzyme replacement therapy for the treatment of patients with Pompe disease (acid α-glucosidase deficiency). It will be available as a 100 mg powder for concentrate for solution for infusion [15].
Apr 21
Target action date (PDUFA) from US FDA extended by 3 months to August 18, 2021 [14]
Mar 21
Granted EAMS status in the UK for treatment of late- and infantile-onset Pompe disease for patients who have already received enzyme replacement therapy with alglucosidase alfa [13].
Nov 20
Awarded Priority Review from US FDA. It has a target action date (PDUFA) of May 18, 2021.[12]
Oct 20
The EMA has accepted for review the MAA for avalglucosidase alfa, for long-term enzyme replacement therapy for the treatment of patients with Pompe disease (acid α-glucosidase deficiency) [11].
Sep 20
Awarded Promising Innovative Medicine (PIM) designation in UK [10]
Apr 20
Company expects to submit regulatory filings in 2020 [9].
May 19
NCT02782741 active, not recruiting, estimated primary completion now Mar 2020 [8].
Jun 18
NCT02782741 remains at recruiting, estimated primary completion now Nov 2021 [7].
May 18
Sanofi pipeline updated, filings now planned for 2020 [6].
Mar 18
Filings planned for 2019 [5].
Feb 18
Has orphan drug status in EU. Will be filed using centralised procedure [4]
Category
A second generation recombinant human alglucosidase alfa product
The overall prevalence has been estimated at 1 in 40,000, with 1 in 138,000 for the infantile form and 1 in 57,000 for the adult form [1].
Late-onset glycogen storage disease type II (Pompe disease) in children and adults
Intravenous infusion
Further information
Yes
Trial or other data
Nov 21
PIII COMET Study (n=100) reports avalglucosidase alfa was non-inferior to alglucosidase alfa at week 49 in terms of improvement in respiratory function, (least-squares mean improvement in upright FVC% predicted of 2.89% vs. 0.46%, respectively) [20].
Nov 20
Sanofi say that avalglucosidase alfa is specifically designed to deliver more GAA enzyme into the lysosomes of the muscle cells and trial results are encouraging. [12]
Oct 16
Sanofi initiates the PIII COMET study of GZ 402666 in patients with glycogen storage disease (Pompe disease) type II (NCT02782741). The study will evaluate the efficacy and safety of repeated biweekly IV infusions of GZ 402666 (20 mg/kg) in comparison with alglucosidase alfa in treatment-naïve patients with late-onset Pompe disease. Primary outcome measure for the trial will be change in percent predicted forced vital capacity (%FVC). The randomised, double-blind study is enrolling approximately 96 patients, aged 3 years and older, in the US, Belgium, Denmark, France, the Netherlands, Sweden and the UK [2].
Collection of primary outcome data in the PIII COMET study (NCT02782741) is expected to complete Oct 20 [3].