2020 End of the Year Summary Report-2 FDA Approved 13 New Large Molecule Drugs and 2 New Gene Therapy Products

Lyophilized powder for intravenous infusion

LM

Patients with active thyroid eye disease received TEPEZZA by intravenous infusions (10 mg/kg for first infusion and 20 mg/kg for the remaining 7 infusions) or placebo infusions every 3 weeks for a total of 8 infusions.

The main outcome measure was the proptosis responder rate at week 24 defined as the percentage of patients with ≥2 mm reduction in proptosis in the study eye from baseline, without deterioration in the non-study eye (≥2 mm increase).”

intravenous infusion

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Trial 1 enrolled patients with episodic migraine. VYEPTI or placebo was administered by intravenous infusion every 3 months for 12 months; however, the primary endpoint was measured at 12 weeks.

Trial 2 enrolled patients with chronic migraine. VYEPTI or placebo was administered by intravenous infusion every 3 months for 6 months; the primary endpoint was measured at 12 weeks.

The primary efficacy endpoint for both trials was the change from baseline in mean monthly migraine days over Months 1-3. Secondary endpoints included the percentages of patients with 50% or greater and 75% or greater reductions from baseline in monthly migraine days over Months 1-3.”

intravenous infusion

LM

Intravenous injection

LM

Patients received TRODELVY 10 mg/kg intravenously on Days 1 and 8 of a 21-day treatment cycle. Tumor imaging was obtained every 8 weeks, and treatment continued until disease progression or intolerance to the therapy.

Major efficacy outcome measures were investigator assessed ORR using RECIST 1.1 and duration of response.”

Intravenous injection

LM

Intravenous 

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Patients received MONJUVI 12 mg/kg intravenously in combination with lenalidomide (25 mg orally on Days 1 to 21 of each 28-day cycle) for maximum of 12 cycles, followed by MONJUVI as a monotherapy until disease progression or unacceptable toxicity.

Efficacy was established based on best overall response rate, defined as the proportion of complete and partial responders, and duration of response as determined by an IRC using the International Working Group Response Criteria.”

Intravenous injection

LM

The primary endpoint was ORR, defined as the proportion of patients with a PR or better, as assessed by an IRC based on the IMWG Uniform Response Criteria for Multiple Myeloma.”

Intravenous injection

Gene Therapy

Trial 1 was a multicenter, 2 period, dose-finding trial. During the initial, double blind period (first 4 weeks), which evaluated the acute safety of VILTEPSO, patients were randomized to VILTEPSO or placebo. All patients were then re-randomized and received 20 weeks of open-label VILTEPSO 40 mg/kg once weekly (0.5 times the recommended dosage) or 80 mg/kg once weekly. The primary efficacy endpoint was the change from baseline in the dystrophin protein level (measured as % of normal) at Week 25.

Trial 2 was a multicenter, parallel-group, open-label, dose-finding trial. Patients were assigned to receive intravenous VILTEPSO 40 mg/kg once weekly (0.5 times the recommended dose) (N=8) or 80 mg/kg once weekly (N=8) for 24 weeks.”

Subcutaneous injection

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In both trials, patients were randomized to and received ENSPRYNG or placebo. In Trial 2, all patients were also receiving either concurrent azathioprine, oral corticosteroids, or mycophenolate mofetil during the trial.

The primary efficacy endpoint was the time to the onset of the first adjudicated relapse evaluated by a blinded Clinical Endpoint Committee (CEC).”

Subcutaneous injection

LM

Enrolled treatment-naïve adult patients with GHD were randomized to once-weekly SOGROYA or placebo for a total of 34-weeks. Additional randomized, open-label arm consisted of patients who received daily somatotropin product.

The efficacy assessment at week 35 was based on the reduction of truncal fat percentage as assessed by dual X-ray absorptiometry.”

Intravenous injection

LM

The primary efficacy endpoint was 28-day mortality. The primary analysis population included all subjects who were randomized and concurrently eligible to receive either INMAZEB or the investigational control during the same time period of the trial.”

subcutaneous injection

Gene Therapy

indicated for the treatment of primary hyperoxaluria type 1 (PH1) to

lower urinary oxalate levels in pediatric and adult patients.”

Trial 1 was a randomized, double blind, placebo-controlled trial that enrolled patients 6 years of age and older with PH1 and an estimated glomerular filtration rate (eGFR) ≥30 mL/min/1.73 m2. Patients received 3 loading doses of 3 mg/kg OXLUMO or placebo administered once monthly followed by quarterly maintenance doses of 3 mg/kg OXLUMO or placebo. The primary endpoint was the percent reduction from baseline in 24 hour urinary oxalate excretion averaged over Months 3 through 6.

Trial 2 was a single arm trial in 16 patients <6 years of age with PH1 and an eGFR >45 mL/min/1.73 m2 for patients ≥12 months of age or a normal serum creatinine for patients <12 months of age. All patients received OXLUMO. The primary endpoint was the percent reduction from baseline in the spot urinary oxalate:creatinine ratio averaged over Months 3 through 6.”

Intravenous injection

mAb

patients with relapsed or refractory high-risk neuroblastoma in the bone or

bone marrow who have demonstrated a partial response, minor response, or

stable disease to prior therapy”

Patients received DANYELZA 9 mg/kg/cycle administered as three separate intravenous infusions of 3 mg/kg (on Days 1, 3 and 5) in the first week of each cycle and GM-CSF subcutaneously at 250 µg/m2/day on Days -4 to 0 and at 500 µg/m2/day on Days 1 to 5.

The major efficacy outcome measure was overall response rate (ORR) as determined by independent pathology and imaging review according to the revised International Neuroblastoma Response Criteria (INRC) and duration of response (DOR), as determined by independent pathology and imaging review.”

Intravenous injection

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IV Injection

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