TEPEZZA is the first and only FDA-approved medicine for the treatment of TED, a serious, progressive and vision-threatening rare autoimmune disease that is associated with proptosis (eye bulging), diplopia (double vision), blurred vision, pain, inflammation and facial disfigurement. TEPEZZA is a fully human monoclonal antibody (mAb) and a targeted inhibitor of the insulin-like growth factor-1 receptor (IGF-1R) that is administered to patients once every three weeks for a total of eight infusions.
“Today is a great day for people living with Thyroid Eye Disease, a rare, vision-threatening disease that previously had no FDA-approved treatment options,” said Timothy Walbert, chairman, president and chief executive officer, Horizon. “The TED community has gone far too long without an FDA-approved therapy, and we are grateful to the people living with TED and physicians who partnered with us on the clinical development program that led to today’s approval of TEPEZZA. This also marks the early approval of Horizon’s first Biologics License Application – a key step in our evolution to an innovation-focused biopharma company, developing new medicines for debilitating diseases with few or no treatment options.”
The FDA approval of TEPEZZA comes ahead of the Prescription Drug User Fee Act (PDUFA) goal date of March 8, 2020. The medicine received Priority Review, Orphan Drug, Fast Track and Breakthrough Therapy designations from the FDA.
“The FDA approval of TEPEZZA is momentous for the TED community and has the potential to change the treatment paradigm for TED – providing new hope for people who are living with this horrible, vision-threatening disease,” said Raymond Douglas, M.D., Ph.D., director of the Orbital and Thyroid Eye Disease Program, Cedars-Sinai Medical Center and co-principal investigator of the TEPEZZA Phase 3 confirmatory clinical trial. “Today’s news brings forward a medicine for patients that targets the underlying biology of the disease and has been shown to significantly improve eye bulging and double vision, which are the most debilitating aspects of the disease.”
“TEPEZZA is a much-needed breakthrough for a community of people who have historically had to struggle in pain as their symptoms progress – risking permanent damage to their eyes and making it extremely difficult to go about their daily lives,” said Jeff Todd, president and chief executive officer, Prevent Blindness. “This approval is meaningful to our organization because we are committed to helping patients with vision impairment and those who are at significant risk.”
The FDA approval of TEPEZZA is supported by a robust body of clinical evidence, including statistically significant, positive results from the Phase 2 clinical study, as well as the Phase 3 confirmatory clinical study OPTIC (Treatment of Graves’ Orbitopathy [Thyroid Eye Disease] to Reduce Proptosis with Teprotumumab Infusions in a Randomized, Placebo-Controlled, Clinical Study). The OPTIC study found that significantly more patients treated with TEPEZZA (82.9%) had a meaningful improvement in proptosis (≥ 2 mm) as compared with placebo patients (9.5%) (p˂0.001) without deterioration in the fellow eye at Week 24. Additional secondary endpoints were also met, including a change from baseline of at least one grade in diplopia (double vision) in 67.9% of patients receiving TEPEZZA compared to 28.6% of patients receiving placebo (p=0.001) at Week 24. In a related analysis of the Phase 2 and Phase 3 clinical studies, there were more patients with complete resolution of diplopia among those treated with TEPEZZA (53%) compared with those treated with placebo (25%). The majority of adverse events experienced with TEPEZZA treatment were graded as mild to moderate and were manageable in the trials, with few discontinuations or therapy interruptions.
Horizon will conduct a post-marketing study to evaluate safety in a larger patient population as was discussed at the Dermatologic and Ophthalmic Drugs Advisory Committee (DODAC) FDA Advisory Committee meeting on December 13, 2019, where the committee voted unanimously (12-0) that TEPEZZA demonstrated a positive benefit risk profile. This study will also evaluate retreatment rates relative to how long patients receive the medicine.
TEPEZZA is expected to be available in the United States in the coming weeks.
Source: Company Press Release