FDA Approves Rockville-based Supernus' Drug-Device Combination for Parkinson's Disease

ROCKVILLE, Md., Feb. 04, 2025 -- Supernus Pharmaceuticals, Inc. (Nasdaq: SUPN), a biopharmaceutical company focused on developing and commercializing products for the treatment of central nervous system (CNS) diseases, announced today that the U.S. Food and Drug Administration (FDA) approved ONAPGO (apomorphine hydrochloride) injection, formerly known as SPN-830, as the first and only subcutaneous apomorphine infusion device for the treatment of motor fluctuations in adults with advanced Parkinson’s disease (PD). Supernus will make ONAPGO available in the second quarter of 2025 with a support team of experts, including a robust nurse education program, and access support at launch.

“Continuous subcutaneous apomorphine infusion already has a proven and established 30-year history in Europe, where it has helped deliver more consistent control of motor fluctuations for thousands of patients,” said Rajesh Pahwa, M.D., Laverne and Joyce Rider Professor of Neurology at the University of Kansas School of Medicine, Director of the Movement Disorder Program at The University of Kansas Health System, and a clinical trial investigator for ONAPGO. “In a clinical trial in Europe, patients treated with ONAPGO experienced a significant reduction in daily OFF time and a similar significant increase in GOOD ON time. Today’s approval of ONAPGO means patients in the U.S. who are not responding well to their current treatment regimen, including levodopa, will now have the option of using a small and lightweight wearable device to deliver a continuous infusion without the need for an invasive surgical procedure.”

The approval is based on results from a Phase 3, 12-week, multicenter, parallel-group, double-blind, randomized, placebo-controlled study (N=107) evaluating the efficacy and safety of ONAPGO. The primary efficacy endpoint was the mean change in total daily OFF time assessed from baseline to the end of the 12-week treatment period based on patient diaries. The key secondary endpoints were the mean change in daily GOOD ON time, which was defined as ON time without troublesome dyskinesia, and Patient Global Impression of Change (PGIC).¹

“ONAPGO represents a novel approach for adults with Parkinson’s disease who are experiencing motor fluctuations,” said Jack Khattar, President and CEO of Supernus Pharmaceuticals. “Supernus’ significant experience in CNS has fueled the success of more than eight widely recognized products in CNS and other therapeutic categories. The addition of ONAPGO demonstrates our continued commitment to developing novel alternatives to manage Parkinson’s disease and other neurological conditions.”

"As Parkinson’s disease progresses, levodopa treatment often becomes less effective at delivering consistent motor control in part due to GI dysmotility, variable absorption of oral medication, and the resulting pulsatile stimulation of dopamine pathways in the brain," said Stuart Isaacson, M.D., Director of Parkinson’s Disease and Movement Disorders Center of Boca Raton, Florida, and a clinical trial investigator for ONAPGO. "With ONAPGO, the continuous infusion of apomorphine directly stimulates postsynaptic dopamine receptors with no metabolic conversion needed. In addition, the subcutaneous delivery of apomorphine bypasses the GI tract and enters the brain, which can allow for more predictable symptom improvement."

“As the motor symptoms of Parkinson’s disease worsen over time, patients report alternating states between ON when their medication is working, and OFF when it’s not working optimally,” said Andrea Merriam, CEO of the Parkinson & Movement Disorder Alliance. “These on-again, off-again changes are disruptive and can happen at any time, which is why consistent daily control of OFF time is key to improving how patients feel and move. For many, continuous treatment options like ONAPGO can help to make days with Parkinson’s more predictable.”

About the Phase 3 Study

During the Phase 3 study, ONAPGO significantly reduced the amount of daily OFF time at 12 weeks from baseline (p=0.0114), with ONAPGO-treated patients (n=53) experiencing a 2.6-hour reduction compared to placebo (n=51) with 0.9 hours. The reduction in daily OFF time was accompanied by a similar significant increase in daily GOOD ON time (2.8 hours for ONAPGO-treated patients compared to 1.1 hours for the placebo group; p=0.0188).¹* In addition, numerically greater improvements in daily OFF time and daily GOOD ON time were seen as early as week 1 and were maintained throughout all measured timepoints. Additionally, ONAPGO-treated patients more frequently reported improvement in their state of general health compared with placebo-treated patients (PGIC: 79% vs. 24%; p<0.0001). The most common adverse events (≥10% incidence) were infusion-site nodule, nausea, somnolence, infusion-site erythema, dyskinesia, headache, and insomnia.¹

About Parkinson’s disease

Nearly one million people in the U.S. and more than 10 million people worldwide are living with Parkinson’s disease, a progressive and chronic neurodegenerative disorder that can cause tremors, muscle rigidity, and difficulty with movement and balance. Patients may also experience dyskinesia, involuntary movements that can significantly interfere with daily activities.² The disease impacts the central nervous system (e.g., the brain and spinal cord) and the peripheral nervous system, the network of nerves that support the limbs and the organs of the body (e.g., GI system including digestion, respiration, heart function, and blood pressure).³ While there is no known cure for PD, there are treatments available to help reduce symptoms.⁴ Patients treated with mainstay regimens may experience periods of GOOD ON time when medication treatment is working well, or OFF time when oral levodopa no longer provides symptom benefit and motor symptoms return.⁵ PD is the second most common neurodegenerative disorder of aging and the most common movement disorder.⁶

USE
ONAPGO is a prescription medicine used to treat motor fluctuations (OFF episodes) in adults with advanced Parkinson’s disease (PD). It is not known if ONAPGO is safe and effective in children.

References

¹ONAPGO. Package insert. Supernus Pharmaceuticals, Inc. *Efficacy results from the analysis of data from the TOLEDO study using the FDA’s preferred methodology, mixed-effects model for repeated measures (MMRM), as required for the submission of the New Drug Application. The results confirmed the statistical significance of the primary outcome.
²Parkinson’s Foundation. Understanding Parkinson’s. Parkinson’s Foundation. 2024. Accessed December 2024. https://www.parkinson.org/understanding-parkinsons.
³American Parkinson Disease Association. Peripheral neuropathy and Parkinson’s disease. 2020. Accessed December 2024. https://www.apdaparkinson.org/article/peripheral-neuropathy-parkinsons-disease/.
⁴World Health Organization. Parkinson disease. 2023. Accessed December 2024. https://www.who.int/news-room/fact-sheets/detail/parkinson-disease.
⁵Isaacson S, Pagan F, Lew M, Pahwa R. Should “on-demand” treatments for Parkinson’s disease OFF episodes be used earlier? Sci Direct. 2022;8:100161.
⁶Mhyre TR, Boyd JT, Hamill RW, Maguire-Zeiss KA. Parkinson’s disease. Subcell Biochem. 2012;65:389–455. doi:10.1007/978-94-007-5416-4_16.

About Supernus Pharmaceuticals, Inc.

Supernus Pharmaceuticals is a biopharmaceutical company focused on developing and commercializing products for the treatment of central nervous system (CNS) diseases.

Our diverse neuroscience portfolio includes approved treatments for attention-deficit hyperactivity disorder (ADHD), dyskinesia in Parkinson’s disease (PD) patients receiving levodopa-based therapy, hypomobility in PD, epilepsy, migraine, cervical dystonia, and chronic sialorrhea. We are developing a broad range of novel CNS product candidates including new potential treatments for hypomobility in PD, epilepsy, depression, and other CNS disorders.

For more information, please visit www.supernus.com.

Forward-Looking Statements

This press release includes forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. These statements do not convey historical information but relate to predicted or potential future events that are based upon management's current expectations. These statements are subject to risks and uncertainties that could cause actual results to differ materially from those expressed or implied by such statements. In addition to the factors mentioned in this press release, such risks and uncertainties include, but are not limited to, the Company’s reporting on preliminary and exploratory open label clinical study on SPN-820, the Company’s ability to sustain and increase its profitability; the Company’s ability to raise sufficient capital to fully implement its corporate strategy; the implementation of the Company’s corporate strategy; the Company’s future financial performance and projected expenditures; the Company’s ability to increase the number of prescriptions written for each of its products and the products of its subsidiaries; the Company’s ability to increase its net revenue; the Company’s ability to commercialize its products and the products of its subsidiaries including ONAPGO; the Company’s ability to enter into future collaborations with pharmaceutical companies and academic institutions or to obtain funding from government agencies; the Company’s product research and development activities, including the timing and progress of the Company’s clinical trials, and projected expenditures; the Company’s ability to receive, and the timing of any receipt of, regulatory approvals to develop and commercialize the Company’s product candidates including SPN-820; the Company’s ability to protect its intellectual property and operate its business without infringing upon the intellectual property rights of others; the Company’s expectations regarding federal, state and foreign regulatory requirements; the therapeutic benefits, effectiveness and safety of the Company’s product candidates including SPN-820; the accuracy of the Company’s estimates of the size and characteristics of the markets that may be addressed by its product candidates; the Company’s ability to increase its manufacturing capabilities for its products, including ONAPGO, and product candidates including SPN-820; the Company’s projected markets and growth in markets; the Company’s product formulations and patient needs and potential funding sources; the Company’s staffing needs; and other risk factors set forth from time to time in the Company’s filings with the Securities and Exchange Commission made pursuant to Section 13 or 15(d) of the Securities Exchange Act of 1934, as amended. The Company undertakes no obligation to update the information in this press release to reflect events or circumstances after the date hereof or to reflect the occurrence of anticipated or unanticipated events.