Insights+: The US FDA New Drug Approvals in August 2020

The US FDA has approved multiple NDAs and BLAs in Aug 2020, leading to treatments for patients and advances in the health care industry. The Center for Drug Evaluation and Research (CDER) and Center for Biologics Evaluation and Research (CBER) have approved 69 novel products so far in 2020, including 10 in Aug 2020. Additionally, last year in 2019, the US FDA has approved 48 novel products. We have compiled a list of a total of 10 new drugs approved by the US FDA in Aug 2020.

1. GSK’s Blenrep (belantamab mafodotin-blmf) Receives the US FDA’s Approval as a Monotherapy Treatment for Multiple Myeloma

Published: Aug 06, 2020 | Tags: Blenrep, US FDA, Monotherapy Treatment, Multiple Myeloma, Antibody Drug Conjugate, Anti-BCMA Therapy, GSK

• The approval is based on a pivotal study, DREAMM-2 assessing Blenrep (2.5 mg/kg, q3w) in 218 patients with relapsed or refractory multiple myeloma who had actively progressing disease that had worsened despite current SoC
• Results: Blenrep demonstrated an ORR (31%), DoR not reached at the 6-mos. analysis, but 73% of responders had a DoR ≥ 6 mos. Continued approval for this indication depends upon verification and description of clinical benefit in confirmatory trials
• Blenrep (belantamab mafodotin-blmf) is an antibody-drug conjugate comprising a humanized anti-B cell maturation antigen (BCMA) monoclonal antibody conjugated to the cytotoxic agent auristatin F. Blenrep is the 1st anti-BCMA therapy approved globally and 5th major medicine approval for GSK in 2020

2. Bayer’s Lampit (nifurtimox) Receives the US FDA’s Approval for Treatment of Chagas Disease in Children

Published: Aug 07, 2020 | Tags: Bayer, Lampit, nifurtimox, Receives, USFDA, Approval, Treatment, Chagas, Disease, Children

• The approval is based on P-III Lampit study assessing nifurtimox in 330 children with chagas disease with serologic evidence of T. cruzi infection in a ratio (2:1) to receive either a 60/ 30 days Lampit treatment regimen, and were followed up for 1yr. after the end of treatment. The company continues the study with a second part (Lampit SECURE) to follow patients for an additional 3yrs.to confirm its efficacy and safety
• Results: showed superiority on nifurtimox 60day arm; Lampit reveals adequate antiprotozoal activity in patients aged 0-17yrs. The indication is approved under accelerated approval based on the number of treated patients who became IgG Ab negative or showed an at least 20% decrease in optical density on two different IgG Ab tests against antigens of T. cruzi
• Lampit is an antiprotozoal agent indicated for pediatric patients aged ≤ 18yrs. and weighing at least 2.5kg for Chagas disease and is currently registered in multiple Latin American countries

3. Trevena’s Olinvyk (oliceridine) Receives the US FDA’s Approval for the Treatment of Central Nervous System

Published: Aug 10, 2020   | Tags: Trevena, Olinvyk, Oliceridine, USFDA Approval, Central Nervous System

• The approval is based on P-III development program assessing Olinvyk in <1,500 patients with moderate to severe acute pain. The loading dose for all Olinvyk treatment regimens (1.5 mg); demand dose (0.1,0.35,0.5mg); and supplemental dose (0.75 mg)
• Result: Olinvyk demonstrated rapid analgesic efficacy statistically significant vs. PBO. Patients using the Olinvyk (doses of 0.35 and 0.5 mg) had a statistically significantly greater SPID-48/24 than patients using PBO
• Olinvyk is a new chemical entity approved in adults for the management of acute pain severe enough to require an IV opioid analgesic. Olinvyk’s product availability is expected in Q4’2020 after the issuing of controlled substance schedule by the US Drug Enforcement Administration (DEA)

4. Roche’s Evrysdi (risdiplam) Receives the US FDA’s Approval for SMA in Adults and Children

Published:  Aug 10, 2020 | Tags: Roche, Evrysdi, US, FDA, SMA, Children, Adults

• The US FDA has approved Evrysdi to treat SMA in adults and children ≥ 2mos. The approval is based on two clinical studies designed to represent a broad spectrum of people living with SMA: FIREFISH in symptomatic infants aged 2-7 mos, and SUNFISH in children and adults aged 2-25yrs.
• The two studies demonstrated improvements in motor function in people with varying ages and levels of disease severity, including Types 1, 2, and 3 SMA. The filing of MAA to EMA for the therapy is imminent while the therapy has been filed in Brazil, Chile, China, Indonesia, Russia, South Korea, and Taiwan
• Roche leads the clinical development of Evrysdi as part of a collaboration with the SMA Foundation and PTC Therapeutics and is the only therapy for SMA that can be taken at home. It will be available in the US within 2wks. for direct delivery to patients’ homes through Accredo Health Group

5. NS Phrama’s Viltepso (viltolarsen) Receives the US FDA’s Approval for Patients with Duchenne Muscular Dystrophy Amenable to Exon 53 Skipping Therapy

Published: Aug 12, 2020 | Tags: NS Pharma, VILTEPSO, viltolarsen, Receives, FDA, Approval, Treatment, Duchenne Muscular

• The approval is based on P-II two-period study; Study 1 includes patients aged 4-10yrs. conducted in North America and Study 2 includes boys aged 5-18yrs. conducted in Japan. The continued approval of VILTEPSO may contingent on confirmation of clinical benefit in P-III confirmatory trial
• Results: patients receiving the dose of (80 mg/kg/wk), 100% patients showed an increase in dystrophin levels; 88% of patients showed dystrophin levels of 3% or greater than normal; after 20-24wks. of treatment, increment in dystrophin expression (6% vs 0.6%)
• Viltepso has received PR designation, RPD, FT & ODD, prior to its  FDA’s approval. In Mar’2020, it has received MHLW’s approval, prior to which it received SAKIGAKE, ODD, and designation of conditional early approval system

6. Roche’s Enspryng (satralizumab-mwge) Receives the US FDA’s Approval for Neuromyelitis Optica Spectrum Disorder

Published:  Aug 17, 2020 | Tags: Roche, Enspryng, US, FDA, NOSD, SAkuraStar, SAkuraSky

• The approval is supported by P-III SAkuraStar and SAkuraSky studies involves assessing Enspryng (120mg, SC, q4w) as a monothx. & as an add-on therapy to baseline IST vs PBO in 95 & 83 patients aged 20-70 & 13-73yrs. in a ratio (2:1) & (1:1) administered at week 0,2 & 4 in patients with NMOSD respectively
• The studies demonstrated robust efficacy and a favorable safety profile in adults with AQP4 Ab positive NMOSD. relapse-free patients @96wks. (76.5% & 91.1% vs 41.1% & 56.8%)
• Enspryng is the first and only FDA-approved SC treatment option for AQP4 Ab positive NMOSD that can be self-administered by a patient or a caregiver every four weeks. The therapy is mAb targeting IL-6 that utilizes recycling antibody technology and will be available in the US in two wks.

7. Recordati Rare Diseases’ Cystadrops (Cysteamine Ophthalmic Solution) Receives the US FDA’s Approval for the Treatment of Ocular Manifestations of Cystinosis

Published: Aug 25, 2020 | Tags: Recordati Rare Diseases, CYSTADROPS, Cysteamine Ophthalmic), USFDA Approval, Ocular Manifestations, Cystinosis

• The approval is based on two clinical trials, a P-III study (n=15) and a P-I/IIa study (n=8) assessing Cystadrops at a median frequency of four times per day
• Result: P-III study showed a 40% reduction in the IVCM total score across all corneal layers from baseline to 90 days and P-I/IIa study demonstrated a 30 % decrease in IVCM total score that was maintained for the 5 year study period
• Cystadrops (cysteamine ophthalmic solution) 0.37% is a cystine-depleting agent indicated for the treatment of corneal cystine crystal deposits in adults and children with cystinosis. It is the 1st and only FDA-approved cysteamine eye drop formulation     

8. Cassiopea’s Winlevi (Clascoterone Cream) Receives the US FDA’s Approval for the Treatment of Acne Vulgaris

Published: Aug 27, 2020 | Tags: Cassiopea, Winlevi, Clascoterone, USFDA Approval, Acne              

• Winlevi is a first-in-class topical androgen receptor inhibitor approved by the US FDA for the treatment of acne in patients (age ≥12 yrs.) with a new mechanism of action (MOA) after ~ 40 yrs.
• Result:  Winlevi demonstrated treatment success and reductions in acne lesions and was well tolerated when used q2w (in pivotal clinical trials). Additionally, the most frequently observed local skin reaction was mild erythema
• Winlevi (clascoterone cream 1%) is an androgen receptor inhibitor indicated for the topical treatment of acne vulgaris by limiting sebum production and inflammation. It is expected to be available in the US in early 2021

9. Novo Nordisk’s Sogroya (somapacitan-beco) Receives the US FDA Approval for Adult Growth Hormone Deficiency

Published: Aug 28, 2020 | Tags: Novo Nordisk, Sogroya, somapacitan-beco, Receives, FDA, Approval, Adult, Growth Hormone Deficiency

• The approval is based on REAL 1 study assessing Sogroya (10 mg/ 1.5 mL (6.7 mg/mL) vs PBO for 35wks. in adult patients with growth hormone deficiency
• The US FDA has approved the BLA of the therapy for the replacement of endogenous growth hormone in adults with GHD
• Sogroya is a prescription medicine containing GH and is used to treat adults who do not make enough growth hormone. Efficacy and safety in children are not known

10. Innocoll’s Xaracoll (bupivacaine HCl) Receives the US FDA’s Approval for the Acute Postsurgical Pain Relief in Adults Following Open Inguinal Hernia Repair

Published: Aug 31, 2020 | Tags: Innocoll, Xaracoll, Bupivacaine, US, FDA, Approval, Acute Postsurgical Pain Relief

• The approval is based on two P-III study (N=610, XARACOLL Arm N=404; PBO Arm N=206) performed as outpatient surgeries in adults across 39 sites in the US.
• Result: Xaracoll provided statistically significant pain relief through 24 hrs. vs PBO, the primary endpoint for both studies. Median time in study 1 (11hrs. vs 1 hrs.) and in study 2 (6hrs. vs 1 hrs.)
• Xaracoll is a unique, non-injectable drug-device combination in the form of a fully bioresorbable collagen implant containing bupivacaine hydrochloride which is placed directly into the surgical site during surgery and, after placement, releases bupivacaine immediately and over time

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