Named Patient Use Program for UPLIZNA® (inebilizumab-cdon)

Horizon Therapeutics plc Announces Partnership with Clinigen Group to Launch Named Patient Use Program for UPLIZNA

-- Program may enable health care providers to access UPLIZNA for people living with AQP4 antibody positive Neuromyelitis Optica Spectrum Disorder (NMOSD) who meet eligibility criteria --

July 19, 2021

Horizon Therapeutics plc today announced a partnership with Clinigen Group, the global pharmaceutical Products and Services company, to make a Named Patient Use (NPU) Program available for UPLIZNA. The UPLIZNA NPU Program is for adult patients with anti-aquaporin-4 (AQP4) antibody positive NMOSD in countries where UPLIZNA is not approved by the country’s local regulatory authority.

The UPLIZNA NPU Program is designed to provide an avenue for physicians – contingent on meeting eligibility criteria and receiving local health approval – to potentially prescribe UPLIZNA on behalf of patients who live in certain countries where it is not available. UPLIZNA is currently approved in the United States and Japan for the treatment of adults with AQP4 antibody positive NMOSD. In Japan, UPLIZNA is marketed by Mitsubishi Tanabe Pharma Corporation.

Through the NPU Program, Horizon and Clinigen will work together to enable health care providers to access UPLIZNA for adults who meet the eligibility criteria. Health care professionals can inquire for more details about UPLIZNA and the NPU Program by calling the Clinigen customer service team at +44 1932 824 123 (RoW) or +44 1932 824 100 (UK) or emailing MedicineAccess@clinigengroup.com.

NMOSD is a unifying term for neuromyelitis optica (NMO) and related syndromes. NMOSD is a rare, severe, relapsing, neuroinflammatory autoimmune disease that attacks the optic nerve, spinal cord and brain stem.1,2 Approximately 80 percent of all patients with NMOSD test positive for anti-AQP4 antibodies.3

About Horizon
Horizon is focused on the discovery, development and commercialization of medicines that address critical needs for people impacted by rare, autoimmune and severe inflammatory diseases. Our pipeline is purposeful: we apply scientific expertise and courage to bring clinically meaningful therapies to patients. We believe science and compassion must work together to transform lives. For more information on how we go to incredible lengths to impact lives, please visit www.horizontherapeutics.com and follow us on Twitter, LinkedIn, Instagram and Facebook.

About Clinigen
Clinigen Group plc (AIM: CLIN) is a global, specialist pharmaceutical services and products platform focused on providing ethical access to medicines. Its mission is to deliver the right medicine to the right patient at the right time. The Group operates from sites in North America, Europe, Africa, and the Asia Pacific.

Clinigen has more than 1,250 employees across five continents in 16 countries, with supply and distribution hubs and operational centres of excellence in key long-term growth regions. The Group works with 34 of the top 50 pharmaceutical companies, interacting with over 5,000 hospitals across more than 115 countries.

For more information on Clinigen, please visit http://www.clinigen.com.

Forward Looking Statements
This release contains forward-looking statements, including, but not limited to, statements related to the anticipated initiation of the UPLIZNA NPU Program and availability of UPLIZNA under the program to patients outside of the United States and Japan; and business and other statements that are not historical facts. These forward-looking statements are based on Horizon’s current expectations and inherently involve significant risks and uncertainties. Actual results and the timing of events could differ materially from those anticipated in such forward-looking statements as a result of these risks and uncertainties, which include, without limitation, the risk of future supply disruptions; impacts of the COVID-19 pandemic and actions taken to slow its spread; regulatory obligations and oversight, including any changes in the legal and regulatory environment in which Horizon operates and those risks detailed from time-to-time under the caption “Risk Factors” and elsewhere in Horizon’s filings and reports with the SEC. Horizon undertakes no duty or obligation to update any forward-looking statements contained in this release as a result of new information.

About UPLIZNA

UPLIZNA (inebilizumab-cdon) is indicated for the treatment of neuromyelitis optica spectrum disorder (NMOSD) in adult patients who are anti-aquaporin-4 (AQP4) antibody positive.

IMPORTANT SAFETY INFORMATION

UPLIZNA is contraindicated in patients with:

  • A history of life-threatening infusion reaction to UPLIZNA
  • Active hepatitis B infection
  • Active or untreated latent tuberculosis

WARNINGS AND PRECAUTIONS

Infusion Reactions: UPLIZNA can cause infusion reactions, which can include headache, nausea, somnolence, dyspnea, fever, myalgia, rash, or other symptoms. Infusion reactions were most common with the first infusion but were also observed during subsequent infusions. Administer pre-medication with a corticosteroid, an antihistamine, and an anti-pyretic.

Infections: The most common infections reported by UPLIZNA-treated patients in the randomized and open-label periods included urinary tract infection (20%), nasopharyngitis (13%), upper respiratory tract infection (8%), and influenza (7%). Delay UPLIZNA administration in patients with an active infection until the infection is resolved.

Increased immunosuppressive effects are possible if combining UPLIZNA with another immunosuppressive therapy.

The risk of Hepatitis B Virus (HBV) reactivation has been observed with other B-cell-depleting antibodies. Perform HBV screening in all patients before initiation of treatment with UPLIZNA. Do not administer to patients with active hepatitis.

Although no confirmed cases of Progressive Multifocal Leukoencephalopathy (PML) were identified in UPLIZNA clinical trials, JC virus infection resulting in PML has been observed in patients treated with other B-cell-depleting antibodies and other therapies that affect immune competence. At the first sign or symptom suggestive of PML, withhold UPLIZNA and perform an appropriate diagnostic evaluation.

Patients should be evaluated for tuberculosis risk factors and tested for latent infection prior to initiating UPLIZNA.

Vaccination with live-attenuated or live vaccines is not recommended during treatment and after discontinuation, until B-cell repletion.

Reduction in Immunoglobulins: There may be a progressive and prolonged hypogammaglobulinemia or decline in the levels of total and individual immunoglobulins such as immunoglobulins G and M (IgG and IgM) with continued UPLIZNA treatment. Monitor the level of immunoglobulins at the beginning, during, and after discontinuation of treatment with UPLIZNA until B-cell repletion especially in patients with opportunistic or recurrent infections.

Fetal Risk: May cause fetal harm based on animal data. Advise females of reproductive potential of the potential risk to a fetus and to use an effective method of contraception during treatment and for 6 months after stopping UPLIZNA.

Adverse Reactions: The most common adverse reactions (at least 10% of patients treated with UPLIZNA and greater than placebo) were urinary tract infection and arthralgia.

For additional information on UPLIZNA, please see Full Prescribing Information at www.UPLIZNA.com.

References

  1. Ajmera MR, Boscoe A, Mauskopf J, Candrilli SD, Levy M. Evaluation of comorbidities and health care resource use among patients with highly active neuromyelitis optica. J Neurol Sci. 2018; 384:96-103.
  2. What is NMO? Guthyjacksonfoundation.org. www.guthyjacksonfoundation.org/neuromyelitis-optica-nmo/. Accessed April 15, 2021.
  3. Layman’s Guide to NMO. Sumairafoundation.org. https://www.sumairafoundation.org/laymans-guide-to-nmo/. Accessed March 9, 2020.

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