dm+d

Unassigned

New Medicines

Nexviadyme (EU), Nexviazyme (US)Late-onset glycogen storage disease type II (Pompe disease) in children and adults

Information

Nexviadyme (EU), Nexviazyme (US)
New formulation
Sanofi
Sanofi

Development and Regulatory status

Phase III Clinical Trials
Approved (Licensed)
Approved (Licensed)
Yes
Jun 22Approved in the EU [22].
Mar 22MHRA 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 21Following a re-examination of its original opinion, the CHMP confirmed that avalglucosidase alfa is not considered a new active substance [19].
Aug 21Sanofi 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 21Approved in US [16]
Jul 21Sanofi 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 21EMA 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 21Target action date (PDUFA) from US FDA extended by 3 months to August 18, 2021 [14]
Mar 21Granted 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 20Awarded Priority Review from US FDA. It has a target action date (PDUFA) of May 18, 2021.[12]
Oct 20The 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 20Awarded Promising Innovative Medicine (PIM) designation in UK [10]
Apr 20Company expects to submit regulatory filings in 2020 [9].
May 19NCT02782741 active, not recruiting, estimated primary completion now Mar 2020 [8].
Jun 18NCT02782741 remains at recruiting, estimated primary completion now Nov 2021 [7].
May 18Sanofi pipeline updated, filings now planned for 2020 [6].
Mar 18Filings planned for 2019 [5].
Feb 18Has 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 20Sanofi 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 16Sanofi 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].

Evidence based evaluations