LIBTAYO is approved for patients with metastatic basal cell carcinoma (mBCC) previously treated with a hedgehog pathway inhibitor (HHI) or for whom an HHI is not appropriate.

• This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for mBCC may be contingent upon verification and description of clinical benefit¹

Efficacy results for LIBTAYO in patients with mBCC (n=28) previously treated with an HHI¹*

Confirmed objective response rate (ORR)

Duration of response (DOR)

Median DOR was not reached for mBCC
(range: (range: 9.0-23.0+ months)¹ Median time to response was
3.2 months (range: 2.1-10.5)¹

• ^†Of the 132 patients in the safety analysis of Study 1620, 48 patients had mBCC.¹

• Serious ARs occurred in 32% of patients. Serious ARs that occurred in >1.5% (at least 2 patients) were urinary tract infection, colitis, acute kidney injury, adrenal insufficiency, anemia, infected neoplasm, and somnolence¹
• Fatal ARs occurred in 1.5% of patients who received LIBTAYO, including acute kidney injury and cachexia¹
• Permanent discontinuation of LIBTAYO due to an AR occurred in 13% of patients¹
• ARs resulting in permanent discontinuation of LIBTAYO in >1.5% (at least 2 patients) were colitis and general physical health deterioration¹
• Dosage delays of LIBTAYO due to an AR occurred in 34% of patients. ARs which required dosage delay in >2% of patients (at least 3 patients) included blood creatinine increased, diarrhea, colitis, fatigue, headache, pneumonitis, and urinary tract infection¹
• The most common ARs reported in at least 15% of patients were fatigue, musculoskeletal pain, diarrhea, rash, pruritus, and upper respiratory tract infection¹
• The most common Grade 3 or 4 ARs (>2%) were hypertension, colitis, fatigue, urinary tract infection, pneumonia, increased blood pressure, hypokalemia, and visual impairment¹
• The most common (>3%) laboratory abnormality worsening from baseline to Grade 3 or 4 was hyponatremia¹

• Toxicity was graded per National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE) v.4.03.¹
• ^‡Fatigue is a composite term that includes fatigue, asthenia, and malaise.¹
• ^§Musculoskeletal pain is a composite term that includes arthralgia, back pain, myalgia, pain in extremity, musculoskeletal pain, neck pain, musculoskeletal stiffness, musculoskeletal chest pain, musculoskeletal discomfort, and spinal pain.¹
• ^||Rash is a composite term that includes rash maculo-papular, rash, dermatitis, dermatitis acneiform, erythema, rash pruritic, dermatitis bullous, dyshidrotic eczema, pemphigoid, rash erythematous, and urticaria.¹
• ^¶Upper respiratory tract infection is a composite term that includes upper respiratory tract infection, nasopharyngitis, rhinitis, sinusitis, pharyngitis, respiratory tract infection, and viral upper respiratory tract infection.¹
• ^#Dyspnea is a composite term that includes dyspnea and dyspnea exertional.¹
• ^**Hypertension is a composite term that includes hypertension and hypertensive crisis.¹

LIBTAYO was studied in a prospective clinical trial that included patients with mBCC previously treated
with an HHI¹

Study 1620 was an open-label, multicenter, phase 2, nonrandomized study that included 132 patients, of which 48 patients had metastatic BCC that had progressed on HHI therapy, had not had an objective response after 9 months on HHI therapy, or were intolerant of prior HHI therapy. Patients received 350 mg every 3 weeks for 5 cycles of 9 weeks followed by 4 cycles of 12 weeks up to 93 weeks. Treatment continued until progression of disease, unacceptable toxicity, or completion of planned treatment. Tumor response assessments were conducted every 9 weeks (during cycles 1 to 5) and then every 12 weeks (during cycles 6 to 9).^1,2

The primary efficacy endpoint was confirmed ORR by ICR. Secondary endpoints included complete response rate, DOR, and safety and tolerability.^1,2

The study excluded patients with autoimmune disease that required systemic therapy with immunosuppressant agents within 5 years; history of solid organ transplant; prior treatment with anti–PD-1/PD-L1 therapy or other immune checkpoint inhibitor therapy; infection with HIV, hepatitis B or hepatitis C; or Eastern Cooperative Oncology Group Performance Status ≥2.¹

• ORR is determined by the proportion of patients with best objective response of CR or PR based on independent central-reviewed evaluation, as determined by RECIST version 1.1 for radiologic assessments, or by modified WHO Criteria for photographic assessments, or by the composite response criteria for patients assessed by both radiology and photography.²
• CR is defined as disappearance of all target lesions for at least 4 weeks. Nontarget lesions also had to be a CR and there could be no new lesions. Any pathological lymph nodes (whether target or nontarget) must have reduction in short axis to <10 mm (<1 cm). Only includes patients with complete healing of prior cutaneous involvement; patients with locally advanced BCC in the study required biopsy to confirm CR.²
• PR is defined as a decrease of 30% or greater in the sum of the diameters of target lesions, taking as reference the baseline sum of diameters, per RECIST 1.1. PR of externally visible disease is defined as a decrease of 50% or greater in the sum of products of perpendicular longest diameters of target lesions, per WHO Criteria. Nontarget lesions could not have PD, and there could be no new lesions. Responses had to be maintained for at least 4 weeks.²
• AR=adverse reaction; CR=complete response; DOR=duration of response; ICR=independent central review; mBCC=metastatic BCC; ORR=objective response rate; PD=progressive disease; PD-1=programmed death receptor-1; PD-L1=programmed death ligand 1; PR=partial response; RECIST=Response Evaluation Criteria in Solid Tumors; WHO=World Health Organization.