Insights+ Exclusive: The US FDA New Drug Approvals in November 2019

Insights+ Exclusive: The US FDA New Drug Approvals in November 2019

The US FDA’s Center for Drug Evaluation and Research (CDER) and Center for Biologics Evaluation and Research (CBER) have approved 35 new products so far in 2019. In 2018 FDA approved 59 novel products including 42 New Chemical Entities and 17 Biologics while breaking its last year’s record of approvals. 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 new drugs approved by the US FDA in November 2019.

1. Sun Pharmaceuticals’ Absorica Received the US FDA’s Approval for Severe Recalcitrant Nodular Acne  

Published: Nov 05, 2019 | Tags: Absorica, Approval, Recalcitrant, Nodular Acne, Received, Severe, Sun Pharmaceuticals, the US FDA

  • The approval was based on the study involves assessing of Absorica vs another isotretinoin 925 patients aged 12 years and older in patients with severe recalcitrant nodular acne
  • Study results: Mean reduction in nodular lesions (-15.68 vs -15.62); 90% reduction in total nodular lesion count (70% vs 75%)
  • Absorica ((isotretinoin) is a prescription medicine granted patients aged 12 years of age and older treat the most severe form of acne nodular acne and have a boxed warning for embryo-fetal toxicity – contraindicated in pregnancy

  2. RedHill Biopharma’s Talicia Received the US FDA’s Approval for H. pylori in Adults

Published: Nov 04, 2019 | Tags: RedHill Biopharma, Adults, Approval, H. pylori, Received, Talicia, the US FDA

  • The approval was based on two P-III study results and assessed Talicia vs active comparator arm and resulted in eradication of H. pylori infection (84% vs 58%), response rate @13 day (90.3% vs 64.7%), No resistance to rifabutin
  • Talicia has market exclusivity of 8 years in the US under QIDP designation in addition to patent protection extending until 2034
  • Talicia is a combination of two antibiotics amoxicillin and rifabutin and a PPI (omeprazole magnesium) is a delayed-release capsules 10 mg1/250 mg/12.5 mg with fixed-dose approved in the US for H. pylori infection in adults, with expected launch in Q1’20

3. Celgene’s Reblozyl (luspatercept–aamt) Received FDA’s Approval as the First Therapy to Treat Patients with Rare Blood Disorder   

Published: Nov 11, 2019 | Tags: Approval, Celgene, FDA, First Therapy, Luspatercept–aamt, Patients, Reblozyl, Receives, Treat

  • The approval was based on P-III BELIEVE study assessing Reblozyl (1.0 mg/kg, SC) + BSC vs PBO + BSC in 336 patients in a ratio (2:1) required RBC transfusions (6-20 RBC units/ 24wks. with no transfusion-free period greater than 35 days during that period) due to beta-thalassemia
  • The P-III BELIEVE study results: improvement in 1EPs & 2EPs i.e, @13–24 wks. ≥33% reduction in RBC transfusion burden (21.4% vs 4.5%); @37-48wks. transfusion burden reduction of at least 33% (19.6% vs 3.6%); @ 13-24 wks. & 37-48 wks. transfusion burden reduction of ≥50% (7.6% & 10.3% vs 1.8% & 0.9%)
  • Reblozyl is a first-in-class erythroid maturation agent, jointly developed by Celgene and Acceleron and has received FDA’s PR designation with its anticipated availability in 1wk. following the FDA approval in the US  

 4. Aquestive Therapeutics’ Exservan (riluzole) Received the US FDA’s Approval to Treat Amyotrophic Lateral Sclerosis (ALS)   

Published: Nov 13, 2019 | Tags: ALS, Amyotrophic Lateral Sclerosis, Approval, Aquestive Therapeutics, Exservan, Receives, Riluzole, the US FDA, Treat

  • Exservan (riluzole) Oral Film received the US FDA approval and has also received FDA’s ODD in Jan 2019 and has been evaluated to assess PK bioequivalence to the Rilutek
  • The ODD is granted to novel drugs and biologics which are intended for the safe and effective treatment, diagnosis or prevention of rare diseases affecting people less than 200,000 in the US
  • Exservan (50mg) is an oral film formulation of approved glutamate Inhibitor riluzole applied on top of the tongue where it adheres and dissolves

5. Cumberland Pharmaceuticals’ RediTrex (methotrexate) Injection Received the US FDA’s Approval for Rheumatoid Arthritis and Psoriasis

Published: Nov 13, 2019 | Tags: Approval, Cumberland Pharmaceuticals, Injection, Methotrexate, Psoriasis, Receives, Reditrex, Rheumatoid Arthritis, the US FDA

  • The approval of RediTrex was done post number of communications with the FDA and several amendments to the NDA submitted to the FDA in late 2018
  • Cumberland will launch two injectable methotrexate product lines within the US targeted to treat active rheumatoid arthritis, juvenile idiopathic arthritis, and severe psoriasis
  • RediTrex (methotrexate) Injection is targeted for adult and pediatric patients with rheumatoid arthritis and adults with psoriasis. Additionally, the company has multiple products in a portfolio focusing on hospital acute care and gastroenterology

6. BeiGene’s Brukinsa (zanubrutinib) Received the US FDA’s Accelerated Approval for Patients with Mantle Cell Lymphoma Who Received At least One Prior Therapy  

Published: Nov 15, 2019 | Tags: Accelerated Approval, BeiGene, Brukinsa, FDA, Mantle Cell, Lymphoma One Prior, Therapy, Receives, Zanubrutinib

  • The US FDA’s accelerated approval was based on P-II BGB-3111-206 and P-I/II BGB-3111-AU-003 studies assessed Brukinsa in patients with relapsed or refractory (R/R) MCL
  • The P-II BGB-3111-206 & P-I/II BGB-3111-AU-003 studies result: ORR (84% & 84%); CR (59% & 22%, FDG-PET scan required & not required); PR (24% & 62%); DOR (19.5 & 18.5 mos.); median follow up time (18.4 & 18.8 mos.)
  • Brukinsa (320mg, PO) is the only FDA approved BTK inhibitor that showed 100% median occupancy in peripheral blood cells and the only BTK inhibitor with the flexibility to be taken once or twice daily with its anticipated availability in the US in the coming wks.

7. Shionogi’s Fetroja (cefiderocol) Received the US FDA’s Approval for the Treatment of Complicated Urinary Tract Infections  

Published: Nov 15, 2019 | Tags: Approva, Cefiderocol, Complicated, Urinary Tract Infections, FDA, Fetroja, Receives, Shionogi, US

  • The US FDA approval was based on APEKS-cUTI study and assessed Fetroja vs imipenem/cilastatin (IPM/CS) in patients aged ≥18yrs. with cUTI who have limited or no alternative treatment options
  • The pivotal APEKS-cUTI study resulted in higher response rate in microbiological eradication and clinical response at the test of cure (TOC), met its 1EPs (72.6% vs 54.6%), SAEs (4.7% vs 8.1%)
  • Fetroja is the first approved antibiotic that functions as a siderophore, act by penetrating the cell wall of gram-negative bacteria and overcome the resistance mechanism and binds to the ferric iron to gain additional cell entry. Fetroja has received FDA’s QIDP, BT and FT designation with its expected availability in the US in H1’20

8. Novartis’ Adakveo (crizanlizumab) Received the US FDA’s Approval for Reducing the Frequency of VOCs in Sickle Cell Disease  

Published: Nov 18, 2019 | Tags: Adakveo, Approval, Crizanlizumab, FDA, Frequency, Novartis, Receives, Reducing Sickle Cell, Disease, US, VOCs

  • The US FDA’s approval was based on SUSTAIN study and assessed Adakveo (5mg/kg, 2.5mg/kg) vs PBO in 198 patients in a ratio 1:1:1 aged ≥ 16yrs. with any genotype of sickle cell disease and a history of 2-10 VOCs/ pain crises in the previous 12 months
  • The SUSTAIN study results: 45% reduction in annual rate of VOCs (1.63 vs 2.98); 42% reduction in the annual rate of days hospitalized (4 vs 6.87days); patients not experiencing VOCs (36% vs 17%); median time to first VOC (4.1 vs 1.4 mos.)
  • Adakveo (SEG101, 5 mg/kg) is a first & only biologic targeting P-selectin, thus blocks the interaction b/w endothelial cells, platelets, red blood cells, and leukocytes and is expected to be available in the coming weeks

9. Alnylam’s Givlaari (givosiran) Received the US FDA’s Approval as the First Therapy for Acute Hepatic Porphyria

Published: Nov 21, 2019 | Tags: Acute Hepatic, Porphyria, Alnylam, Approval, FDA, First Therapy, Givlaari, Givosiran, Receives, US

  • The US FDA approval was based on P-III ENVISION study and assessed Givlaari vs PBO in 94 patients with AHP at 36 sites across 18 countries. Givlaari got FDA’s approval in less than four months following its NDA acceptance
  • The P-III ENVISION study resulted in 70% fewer porphyria attacks in patients, reduction in intravenous hemin use, reductions in urinary aminolevulinic acid and urinary porphobilinogen. Additionally, in Aug’2019, Alnylam signed a promotional agreement with Ironwood for GIVLAARI to raise awareness of AHP among gastroenterologists and other HCPs in the US
  • Givlaari is the world’s first approved GalNAc-conjugate RNA therapeutic targeting aminolevulinic acid synthase 1 (ALAS1) and has received FDA’s BT & ODD and EMA’s PRIME & ODD with its anticipated availability in the US by the year-end

10. SK Life Sciences’ XCOPRI (cenobamate tablets) Received the US FDA’s Approval for Partial-Onset Seizures in Adults  

Published: Nov 21, 2019 | Tags: Adults, Approval, Cenobamate Tablets, Partial-Onset, Receives, Seizures, Sk Life Sciences, US FDA, Xcopri

  • The approval was based on two global studies (Study 013 and Study 017) and assessed XCOPRI vs PBO and demonstrated significant reductions in seizure frequency vs PBO at all doses studied
  • Study 013 results: XCOPRI (200 mg/day) (56% vs 22%), zero seizures (28% vs 9%); Study 017: XCOPRI (100 mg/day). During maintenance phase of 013, 017 reduction in median seizures @ 100/200/400 mg/day (36%, 55%, 55% vs 24 %); (4%, 11%, 21% vs 1%)
  • XCOPRI (cenobamate tablets) is an anti-epileptic drug targeted for partial-onset seizures in adults with expected availability in the US in Q2’2020. Additionally, in 2019 SK biopharma signs an exclusive license agreement with Arvelle Therapeutics to develop and commercialize in EU

11. Global Blood’s Oxbryta (Voxelotor) Received the US FDA’s Accelerated Approval for Treatment of Sickle Cell Disease

Published: Nov 21, 2019 | Tags: Accelerated Approval, Global Blood, Oxbryta, Receives, The US FDA, Treatment, Voxelotor

  • The approval was based on P-III HOPE (Hemoglobin Oxygen Affinity Modulation to Inhibit HbS PolymErization) study assessed Oxbryta vs PBO in 274 patients of 12 years and older with SCD
  • The study resulted in achieving greater than 1g/dL increase in hemoglobin (51.1% vs 6.5%), Published in NEJM Jun’2019 
  • Oxbryta (bid) is an orally administered drug the first approved treatment that directly inhibits sickle hemoglobin polymerization for SCD and has is reviewed under Priority Review received BT designation FT, ODD and Rare Pediatric Disease designations for the treatment of patients with SCD