dm+d

Unassigned

New Medicines

UpliznaNeuromyelitis optica spectrum disorder (NMOSD) in adults who are anti-aquaporin-4-seropositive

Information

Uplizna
New molecular entity
Horizon Therapeutics
Horizon Therapeutics

Development and Regulatory status

None
Approved (Licensed)
Launched
Yes
Yes
May 22Approved in EU as monotherapy for the treatment of adult patients with neuromyelitis optica spectrum disorder (NMOSD) who are anti-aquaporin-4 immunoglobulin G seropositive (AQP4-IgG+) [21].
Feb 22First European launch will be Germany in Q2 2022, as well as other international markets in the coming years [20].
Nov 21Company reports plans for globalisation starting with 2022 launch of Uplizna in Europe pending approval in Q1 2022 [19]
Nov 21Recommended for EU approval by CHMP “as monotherapy for the treatment of adult patients with neuromyelitis optica spectrum disorders (NMOSD) who are anti-aquaporin 4 immunoglobulin G (AQP4-IgG) seropositive”. Uplizna will be available as a 100 mg concentrate for solution for infusion [16].
Feb 21Horizon Therapeutics acquire Viela Bio [17].
Jan 21Filed in EU via centralised procedure [14].
Jun 20Licensed by FDA. Launch expected June 2020 [13].
Apr 20Following advice received from the company, NICE will announce timelines for an appraisal at a later date [11].
Mar 20According to its latest annual report, Viela believes it can effectively commercialise inebilizumab in the US with a focused commercial team targeting a small number of medical centres of excellence that treat approximately 70% of the estimated 10,000 patients with NMOSD. In addition to a collaboration with Hansoh Pharma and MTPC (for co-development and commercialisation in selected Asian countries), Viela is evaluating potential additional partnerships to pursue regulatory approval and commercialisation of inebilizumab in NMOSD in other geographic regions outside of the US. No mention of plans as yet to file in the EU [12].
Nov 19Viela Bio announced that the PDUFA action date of June 11 2020 has been assigned by the US FDA [10].
Sep 19The FDA has accepted inebilizumab for review for the treatment of neuromyelitis optica spectrum disorder [8].
Apr 19Granted Breakthrough Therapy designation in the US based on results from the pivotal phase II/III N-MOmentum trial [7].
Mar 19Viela Bio announced plans to file a licensing application with the US FDA in Q2 19 based on results from the PIIb pivotal trial of inebilizumab in pts with NMOSD.[6]
Dec 18Inebilizumab listed in Viela Bio Pipeline as PIIb [5]
Feb 18AstraZeneca announced that its global biologics research and development arm, MedImmune, is spinning out six molecules from its early-stage inflammation and autoimmunity programmes into an independent biotech company, Viela Bio, one of which is inebilizumab for the treatment of neuromyelitis optica [4].

Category

A humanised immunoglobulin G 1 kappa (IgG1κ) monoclonal antibody directed against B-cell antigen CD19 [1].
Neuromyelitis Optica is a rare disorder affecting eyes and spinal cord leading loss of vision, mobility, and sensation. It affects about 0.4 in 10,000 people in the EU equivalent to a total of around 20,000 people.
Neuromyelitis optica spectrum disorder (NMOSD) in adults who are anti-aquaporin-4-seropositive
Intravenous

Further information

Yes

Trial or other data

Nov 21New post hoc analysis of N-MOmentum PII/III pivotal trial has been published in Multiple Sclerosis and Related Disorders journal, showing that prior rituximab exposure did not impact the efficacy of Uplizna, and that Uplizna demonstrated comparable efficacy to trial participants without prior exposure to rituximab [18].
Sep 19PII/III N-MOmentum trial (NCT02200770; n=230) is published in the Lancet; it reports that compared with placebo, inebilizumab reduced the risk of a neuromyelitis optica spectrum disorder (NMOSD) attack; 21/174 (12%) of inebilizumab patients had an attack vs. 22 (39%) of 56 patients on placebo (HR 0.272, 95% CI 0.150–0.496; p<0.0001) [9].
Jan 19Positive topline results from the PIIb N-MOmentum trial (NCT02200770) reported. Pts on inebilizumab monotherapy saw a 77% decrease in the risk of developing NMOSD attack vs. those on placebo. Secondary analysis showed a decrease in worsening of disability in pts receiving the drug and safety and tolerability were acceptable and consistent with previous data. Full data are expected to be presented at the American Academy of Neurology meeting in May.[6]
Oct 18Recruitment for NCT02200770 trial completed [3].
Jan 15Recruitment started for a multicentre, multinational, randomised double-blind PII/III trial (N-MOmentum; NCT02200770) with optional open-label period to evaluate the efficacy and safety of inebilizumab (MEDI-551) in adults with neuromyelitis optica or neuromyelitis optica spectrum disorders (NMO/NMOSD). Eligible subjects (n=252 planned) will first enter a randomised-controlled period (RCP) of up to 197 days where they will be randomised in a 3:1 ratio to receive either IV inebilizumab or placebo, with the primary outcome measure the time to onset of first NMO/NMOSD attack. Attacks will be evaluated by the investigator and confirmed against the attack criteria by an independent Adjudication Committee (AC). Subjects will then be given the option to enrol into an open label period of inebilizumab treatment for at least 12 months and maximum 3 years after the last subject has been enrolled. Any subjects discontinuing the trial at any point will enter a safety follow-up period of 12 months. Estimated primary completion date is Nov 2019 [2].

Evidence based evaluations