2020 End of the Year Summary Report-2 FDA Approved 13 New Large Molecule Drugs and 2 New Gene Therapy Products BLA Regulatory, LLC (Gaithersburg, MD) provides the following summary of 2020 FDA approved new large molecule (LM) drugs and gene therapy products. Reviewers/Readers are suggested to open FDA links for detailed information and accuracy. Number of approved LM drugs and Gene Therapies in sub-categories Therapeutic Area Formulation Regulatory Program Oncology 6 IV Solution 11 Breakthrough Therapy 2 Genetic disorder 3 Subcutaneous Solution 3 Fast Track 1 Neurology 2 Lyophilized powder 1 Orphan Drug 11 Infection 2 Priority Review 3 Metabolic disease 1 Ophthalmology 1 Page 1 of 6 If re-sharing and re-printing, please indicate BLA as the source of this summary. Number New Drug Sponsor indication Drug Regulatory Unique clinical design Details Approve formulation target special Date program 1 TEPEZZA Horizon Treatment of IGF-1R Priority "There were two randomized, double-masked, placebo-controlled trials that FDA link 1/21 (teprotumum Pharma Thyroid Eye Review, provided data for approval of TEPEZZA. ab-trbw) Ireland, Disease Fast Track, Ltd. Breakthrou Patients with active thyroid eye disease received TEPEZZA by intravenous Lyophilized gh Therapy infusions (10 mg/kg for first infusion and 20 mg/kg for the remaining 7 powder for Designation infusions) or placebo infusions every 3 weeks for a total of 8 infusions. intravenous and Orphan infusion Drug The main outcome measure was the proptosis responder rate at week 24 designation defined as the percentage of patients with ≥2 mm reduction in proptosis in LM the study eye from baseline, without deterioration in the non-study eye (≥2 mm increase)." 2 VYEPTI Lundbeck The preventive CGRP N/A "The efficacy and safety of VYEPTI were evaluated in two similar, FDA Link 2/21 (eptinezuma Seattle treatment of randomized, multicenter, placebo-controlled clinical trials. b-jjmr) BioPharm migraine in adults aceuticals, Trial 1 enrolled patients with episodic migraine. VYEPTI or placebo was intravenous Inc. administered by intravenous infusion every 3 months for 12 months; infusion however, the primary endpoint was measured at 12 weeks. LM 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." 3 SARCLISA® Sanofi The treatment of CD38 Orphan The efficacy and safety of SARCLISA in combination with pomalidomide and FDA Link 3/2 (isatuximab- Aventis adult patients with Drug design low-dose dexamethasone were evaluated in a multicenter, randomized, irfc) US LLC multiple myeloma ation open-label trial in patients who had had received at least two prior therapies who have including lenalidomide and a proteasome inhibitor. Patients were randomized intravenous received at least in a 1:1 ratio to receive either SARCLISA in combination with pomalidomide infusion two prior and low-dose dexamethasone or pomalidomide and low-dose therapies dexamethasoneTreatment was administered in both groups in 28-day cycles LM including until disease progression or unacceptable toxicity. The primary endpoint was lenalidomide and progression-free survival (PFS). PFS results were assessed by an a proteasome Independent Response Committee based on central laboratory data for M- inhibitor protein and central radiologic imaging review using the International Myeloma Working Group (IMWG) criteria. Page 2 of 6 If re-sharing and re-printing, please indicate BLA as the source of this summary. Number New Drug Sponsor indication Drug Regulatory Unique clinical design Details Approve formulation target special Date program 4 TRODELVY Immunom The treatment of TACSTD N/A "There was one multicenter, single-arm, trial that enrolled patients with FDA Link 4/22 ™ edics, Inc adult patients with 2 metastatic triple-negative breast cancer (mTNBC) who had received at least (sacituzumab metastatic triple- two prior treatments for metastatic disease. govitecan- negative breast hziy) cancer (mTNBC) Patients received TRODELVY 10 mg/kg intravenously on Days 1 and 8 of a who have 21-day treatment cycle. Tumor imaging was obtained every 8 weeks, and Intravenous received at least treatment continued until disease progression or intolerance to the therapy. injection two prior therapies for Major efficacy outcome measures were investigator assessed ORR using LM metastatic RECIST 1.1 and duration of response." disease. 5 UPLIZNA™ Viela Bio, The treatment of B- Orphan There was one trial of 197 days duration that evaluated the benefits and side FDA Link 6/11 (inebilizumab Inc. neuromyelitis lymphocy Drug effects of UPLIZNA. The trial enrolled adult patients with NMOSD who were -cdon) optica spectrum te designation positive for anti-aquaporin-4 antibody. Patients received at random either disorder antigen UPLIZNA or placebo infusions according to the schedule. Neither the Intravenous (NMOSD) in adult CD19 patients nor the healthcare providers knew which treatment was being given. injection patients who are anti-aquaporin-4 LM (AQP4) antibody positive 6 MONJUVI® MorphoSy The treatment of B- N/A; "The safety and efficacy of MONJUVI were established in an open label, FDA Link 7/31 (tafasitamab- s US Inc. adult patients with lymphocy Orphan multicenter trial in adult patients with relapsed or refractory DLBCL after 1 to cxix) relapsed or te 3 prior systemic therapies. Enrolled patients were not candidates for high refractory diffuse antigen dose chemotherapy followed by autologous stem cell transplantation. Intravenous large B-cell CD19 lymphoma Patients received MONJUVI 12 mg/kg intravenously in combination with LM (DLBCL) not lenalidomide (25 mg orally on Days 1 to 21 of each 28-day cycle) for otherwise maximum of 12 cycles, followed by MONJUVI as a monotherapy until specified, disease progression or unacceptable toxicity. including DLBCL arising from low Efficacy was established based on best overall response rate, defined as the grade lymphoma, proportion of complete and partial responders, and duration of response as and who are not determined by an IRC using the International Working Group Response eligible for Criteria." autologous stem cell transplant (ASCT) Page 3 of 6 If re-sharing and re-printing, please indicate BLA as the source of this summary. Number New Drug Sponsor indication Drug Regulatory Unique clinical design Details Approve formulation target special Date program 7 BLENREP GlaxoSmit The treatment of TNFRSF N/A; "The efficacy and safety of BLENREP were evaluated in a multicenter, open- FDA Link 8/5 (belantamab hKline adult patients with 17 Orphan label trial of patients with relapsed refractory multiple myeloma who had mafodotin- Intellectual relapsed or Drug previously received 3 or more anti-myeloma treatment regimens, and whose blmf) Property refractory multiple designation disease no longer responded to an immunomodulatory agent and a Developm myeloma who proteasome inhibitor. Treatment was administered as an intravenous Intravenous ent Ltd. have received at infusion once every 3 weeks until disease progression or unacceptable injection least 4 prior toxicity. therapies LM including an anti- The primary endpoint was ORR, defined as the proportion of patients with a CD38 monoclonal PR or better, as assessed by an IRC based on the IMWG Uniform Response antibody, a Criteria for Multiple Myeloma." proteasome inhibitor, and an immunomodulator y agent. 8 VILTEPSO Nippon The treatment of DMD Priority "VILTEPSO for the treatment of DMD was evaluated in two clinical trials. FDA Link 8/12 (viltolarsen) Shinyaku Duchenne gene Review, Enrolled patients had a genotypically confirmed DMD diagnosis (a confirmed Co., Ltd. muscular (exon 53 Orphan mutation of the DMD gene that is amenable to exon 53 skipping) and were Intravenous dystrophy (DMD) viltolarse Drug on a stable dose of corticosteroids for at least 3 months prior to enrollment. injection in patients who n target designation have a confirmed site) Trial 1 was a multicenter, 2 period, dose-finding trial. During the initial, Gene mutation of the double blind period (first 4 weeks), which evaluated the acute safety of Therapy DMD gene that is VILTEPSO, patients were randomized to VILTEPSO or placebo. All patients amenable to exon were then re-randomized and received 20 weeks of open-label VILTEPSO 53 skipping. 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." 9 ENSPRYNG Genentec The treatment of Interleuki N/A; "The safety and efficacy of ENSPRYNG were established in two FDA Link 8/14 ™ h, Inc. neuromyelitis n-6 Orphan randomized, placebo- controlled trials of patients with NMOSD. (satralizuma optica spectrum receptor Drug b-mwge) disorder subunit designation In both trials, patients were randomized to and received ENSPRYNG or (NMOSD) in adult alpha placebo. In Trial 2, all patients were also