Why Actimmune, Horizon’s Lead Orphan Product, Has Such Huge Potential


Mar. 31 2016, Updated 9:08 a.m. ET


After the Vidara merger in September 2014, Horizon Pharma (HZNP) gained access to the company’s drug, Actimmune, which has been approved by the FDA (US Food and Drug Administration) for CGD (chronic granulomatous disease) and SMO (severe malignant osteopetrosis). Actimmune fetched $107.4 million for Horizon in fiscal 2015. The company is following a label expansion strategy to widen the base of the target population for the drug.

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Actimmune for Friedreich’s ataxia

Horizon has initiated the Phase 3 trial for Actimmune, which treats FA (Friedreich’s ataxia). The company expects the drug data to be available by December 2016, and the company plans to file Supplemental Biologics License Application by the first quarter of 2017. As the drug holds fast-track designation, it should win FDA approval by 3Q17.

The prevalence for Friedreich’s ataxia is currently ~3,700 cases in the US, but there is no FDA-approved therapy for the disorder.

Actimmune in combination with Opdivo

Horizon has initiated a Phase 1 trial in kidney and bladder cancer. During December 2015, Horizon initiated a Phase 1 dosing trial of Actimmune in combination with Opdivo in advanced solid tumors.

Currently approved PD-L1 (programmed death ligand 1) inhibitor therapies include Merck & Company’s (MRK) Keytruda and Bristol-Myers Squibb’s (BMY) Opdivo. Roche’s (RHHBY) PD-L1 inhibitor therapy, Atezolizumab, is in the pipeline.

Actimmune for CGD and SMO

Actimmune is indicated for “reducing the frequency and severity of serious infections associated with CGD and SMO.” The incidence of CGD and SMO is around 1 in 100,000–200,000 (to 250,000 in newborns).

This FA indication presents the drug with a $500 million–$1 billion peak sales opportunity. With continued label expansion for other indications, Horizon expects the drug to add $800 million–$1.5 billion in net sales by 2020.

To get exposure to Horizon while controlling excessive company-specific risks, investors can choose to invest in the iShares US Pharmaceuticals ETF (IHE). Horizon accounts for 1.7% of IHE’s total holdings.

Continue to the next and final part for a look at the latest addition to Horizon’s stable of orphan drugs.


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