• The US FDA's Center for Drug Evaluation and Research (CDER) and Center for Biologics Evaluation and Research (CBER) have approved 48 new products so far in 2019.
• However, there is a significant increase in multiple diseases while the new approvals are helping and advancing the changes to understand, diagnose, and treat the diseases
• Our team at PharmaShots has compiled a list of a total 11 new drugs approved by the US FDA in March and April 2020

MARCH

1. Novartis Isturisa (osilodrostat) Receives the US FDA's Approval for Cushing's Disease

Published: Mar 09, 2020 | Tags: Approval, Cushing's Disease, FDA, Isturisa, Novartis, Osilodrostat, Received, US

• The approval is based on a study assessing Isturisa {starting dose of 2mg (bid) that could be increased q2w up to 30mg (bid)} in 137 adults with Cushing's disease who either had undergone pituitary surgery that did not cure the disease or were not surgical candidates
• The study resulted that half of the patients had cortisol levels within normal limits @24wks. Following the above study, 71 patients entered into 8wks. a study assessing Isturisa vs PBO which resulted in maintenance of cortisol level (86% vs 30%)
• Isturisa is the first FDA-approved therapy targeting the cortisol overproduction by blocking the enzyme k/a 11-beta-hydroxylase and preventing cortisol synthesis and has received FDA's ODD for the same indication

2. Curium's Pulmotech MAA Receives the US FDA's Approval for Lung Imaging

Published: Mar 24, 2020 | Tags: Approval, Curium, Lung Imaging, Pulmotech, MAA, Received, The US FDA 

• Pulmotech MAA is a single-photon emission agent targeted for lung imaging as an adjunct in the evaluation of pulmonary perfusion, further aiding the evaluation of peritoneovenous shunt patency in adults
• The company offers to purchase vials in either a pack of 5 or in a box of 30 and has also made a similar version of MAA (macroaggregated albumin) in EU with expected commercialization in Apr 2020 in the US
• Technetium Tc 99m Albumin Aggregated Injection is a radioactive diagnostic agent which is targeted for Lung scintigraphy and Scintigraphy of peritoneovenous shunt in adults

3. BMS Zeposia (ozanimod) Receives the US FDA's Approval as New Oral Treatment for Relapsing Forms of Multiple Sclerosis

Published: Mar 26, 2020 | Tags: BMS, Approval, FDA, Multiple Sclerosis, New, Oral, Treatment, Ozanimod, Received, Relapsing, Forms, US, Zeposia

• The approval is based on P-III SUNBEAM & RADIANCE Part B studies assessing Zeposia (0.92 mg, equivalent to 1mg) vs Avonex (interferon beta-1a, qw, IM) in 1,346 & 1,320 patients with RMS for at least 12 & 24mos. treatment period respectively
• Collective results: @1yr. & @2yrs.; reduction in ARR of 48% & 38% (0.18 vs 0.35 & 0.17 vs 0.28); reduction in T1-weighted GdE brain lesions (0.16 vs 0.43 & 0.18 vs 0.37), reduction in number of new or enlarging T2 lesions (1.47 vs. 2.84 & 1.84 vs 3.18) respectively
• Zeposia is an S1P receptor modulator that binds with high affinity to S1P receptors 1 and 5, act by blocking the capacity of lymphocytes to egress from lymph nodes thus reducing the number of lymphocytes in peripheral blood

4. Allergan's Durysta (bimatoprost implant) Receives the US FDA's Approval as the First Intracameral Biodegradable Sustained-Release Implant for Patients with OAG and OHT

Published: Mar 26, 2020 | Tags: Allergan, Approval, Bimatoprost, Implant, Biodegradable, Durysta, FDA, First, Intracameral, OAG, OHT Patients, Received, Sustained-Release, Implant, US

• The approval is based on two P-III ARTEMIS studies (including 8mos. extended follow up) assessing Durysta (10mcg) vs topical timolol drops (bid) in 1,122 patients with OAG & OHT.
• The two P-III ARTEMIS studies resulted in a 30% reduction in IOP from baseline in 12wks. primary efficacy period, meeting the predefined criteria for non-inferiority
• The approval expands the availability of Allergan's EyeCue, a reimbursement service for eye care professionals facilitating the patient benefit verification & PA assistance for Allergan Eye Care products. Durysta is a prostaglandin analog, being evaluated in 5 P-III studies that support its FDA label enhancement & ROW approvals

5. Rockwell's Triferic AVNU Receives the US FDA's Approval to Replace Iron and Maintain Hemoglobin Hemodialysis Patients

Published: Mar 27, 2020 | Tags: Approval, Hemodialysis, Patients, Hemoglobin, Iron, Maintain, Received, Replace, Rockwell, The US FDA, Triferic, Avnu

• The approval is based on the study assessing Triferic in adult patients with hemodialysis-dependent chronic kidney disease and has resulted in steady and consistent bioavailable iron to replace the iron that is lost at every dialysis treatment and hemoglobin is maintained
• The company will launch Evaluation Programs for evaluating Triferic and with an expected launch in Q3'20
• Triferic AVNU (ferric pyrophosphate citrate) is a IV formulation of Triferic and a novel treatment for iron replacement and maintenance of hemoglobin in hemodialysis patients

6. Sanofi's Sarclisa (isatuximab-irfc) Receives the US FDA's Approval for Relapsed Refractory Multiple Myeloma

Published: Mar 27, 2020 | Tags: Approval, FDA, Isatuximab-Irfc, Received, Relapsed, Refractory, Multiple, Myeloma, Sanofi, Sarclisa, US

• The approval is based on P-III ICARIA-MM study Sarclisa (IV, 10mg/kg) + pomalidomide + dexamethasone (pom-dex) vs pom-dex as monothx. in patients with RRMM, prior treated with at least two therapies including lenalidomide and a proteasome inhibitor
• The P-III ICARIA-MM study results: improvement in PFS (11.53 vs 6.47); improvement in ORR (60.4% vs 35.3%)
• Sarclisa is mAb targeting CD38 receptor on MM cells, act by inducing apoptosis & immunomodulatory activity and has received FDA & EMA's ODD with its anticipated availability in the US soon

APRIL

1. AstraZeneca and MSD's Koselugo (selumetinib) Received the US FDA's Approval as the First Therapy for Pediatric Patients with Neurofibromatosis Type 1 Plexiform Neurofibromas

Published: Apr 13, 2020 | Tags: Approval, AstraZeneca, FDA, First, Therapy, Koselugo, MSD, Nf1- Pn, Pediatric, Patients, Received, Selumetinib, US

• The approval was based on P-I/II SPRINT study which assessed Koselugo (bid) as monothx. in pediatric patients aged =2yrs. with NF1-related inoperable PNs. The trial was sponsored by NCI CTEP, conducted under a research and development agreement between NCI and AstraZeneca with additional support from NTAP
• The P-I/II SPRINT study demonstrated 66% ORR in pediatric patients with NF1 PN. The two companies jointly developed & commercialized Koselugo globally and have submitted an MAA to EMA in Q1'20
• Koselugo is a MEK1/2 inhibitor and has received the US FDA's BT designation in Apr'2019, RPD in Dec'2019, ODD in Feb'2018, EMA's ODD in Aug'2018 and Swissmedic's ODD in Dec'2018 for the treatment of pediatric patients with NF1 PN

2. Incyte's Pemazyre (pemigatinib) Received the US FDA's Approval as the Novel Treatment for Adults with Previously Treated, Unresectable LA or Metastatic Cholangiocarcinoma

Published: Apr 17, 2020 | Tags: Incyte, Pemazyre, pemigatinib, Received, the US FDA, Approval, Novel, Treatment, Adults, Previously Treated, Unresectable, LA, Metastatic Cholangiocarcinoma

• The approval was based on P-II FIGHT-202 (NCT02924376) study which involved assessing of Pemazyre (13.5 mg, qd) in adults with previously treated, locally advanced or metastatic cholangiocarcinoma with documented FGFR2 fusion or rearrangement
• The study has three cohorts: Cohort A (FGFR2 fusions or rearrangements), Cohort B (other FGF/FGFR genetic alterations), or Cohort C (no FGF/FGFR genetic alterations) and resulted in OR 36%, mDOR 9.1 mos.
• Pemazyre is an oral inhibitor of FGFR isoforms 1, 2 and 3 and has also received the US FDA's BT Designation and ODD to treat patients with previously treated advanced/metastatic or unresectable FGFR2 translocated cholangiocarcinoma and for cholangiocarcinoma respectively

3. Seattle's TUKYSA (tucatinib) Received the US FDA's Approval for Advanced Unresectable or Metastatic HER2-Positive Breast Cancer

Published: Apr 17, 2020 | Tags: Advanced, Approval, Breast Cancer, Metastatic, Her2-Positive, Received, Seattle, The US FDA, Tucatinib, Tukysa, Unresectable

• The approval was based on HER2CLIMB study assessing TUKYSA in combination with trastuzumab and capecitabine vs trastuzumab & capecitabine monothx the ratio (2:1) in 612 patients with 1L+ Anti-HER2 therapies in the metastatic setting either monothx or in combination, trastuzumab, pertuzumab, and ado-trastuzumab emtansine (T-DM1)
• The study resulted in a 46% reduction in the risk of cancer progression or death (PFS), OS 34%, objective response (40.6% vs 22.8%); for patients with brain metastases reduction in PFS is 52%. Published in NEJM Dec 2019
• TUKYSA (tucatinib, PO) is tyrosine kinase inhibitor of the HER2 protein and has also received the US FDA's BT Designation and Priority Review for and reviewed this application for approval under the Real-Time Oncology Review (RTOR) pilot program

4. Immunomedics Trodelvy Received the US FDA's Accelerated Approval for 2L Patients with Metastatic Triple-Negative Breast Cancer (TNBC)

Published: Apr 22, 2020 | Tags: Immunomedics, Trodelvy, Received, the US FDA, Accelerated, Approval, 2L, Patients, Metastatic Triple-Negative Breast Cancer, TNBC

• The approval followed the P-II study which involved assessing of Trodelvy in 108 adults with TNBC who had previously received a median of three prior systemic therapies in the metastatic setting (range: 2-10)
• The study resulted in ORR 33.3%, mDOR 7.7 mos. Additionally, the P-III ASCENT study (N=500) is halted by the independent Data Safety Monitoring Committee (DSMC) for compelling evidence of efficacy across multiple endpoints with expected results in mid-2020
• TRODELVY (sacituzumab govitecan-hziy) is a novel ADC targeting the Trop-2 antigen approved by the US FDA for relapsed or refractory metastatic TNBC and is currently being evaluated as a treatment for eight hard-to-treat solid cancers

5. UroGen's Jelmyto Received the US FDA's Expedited Approval as a Novel Non-Surgical Treatment for Patients with Low-Grade Upper Tract Urothelial Cancer

Published: Apr 15, 2020 | Tags: UroGen, Jelmyto, Received, the US FDA, Expedited, Approval, Novel, Non-Surgical, Treatment, Patients, Low-Grade Upper Tract Urothelial Cancer

• The approval was based on P-III OLYMPUS trial results assessing Jelmyto in 74 adults with LG UTUC to evaluate its safety, tolerability, and tumor ablative effect across the US and Israel
• The study resulted in CR 58%, Kaplan-Meier analysis estimated 12-month durability at 84%, mDOR has not been reached, no treatment-related deaths occurred
• Jelmyto (mitomycin for pyelocalyceal solution) is a mitomycin, has also received the US FDA's ODD, Fast Track, and Breakthrough Therapy Designations for the treatment of LG UT

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