Insights+: The US FDA New Drug Approvals in September and October 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 11 new drugs approved by the US FDA in September and October 2019.

1. Xeris’ GVOKE (glucagon) Received FDA’s Approval as The First Ready-To-Use Stable Liquid Glucagon for Severe Hypoglycemia

Published: Sept 11, 2019 | Tags: Approval, FDA, Glucagon, Gvoke, Ready-To-Use, Stable, Liquid, Glucagon, Receives, Severe Hypoglycemia, Xeris

• The approval was based on three P-III studies (NCT02656069, NCT03091673, NCT03439072) assessing GVOKE vs conventional glucagon emergency kits in adults and children aged ≥2yrs. with T1D
• The studies demonstrated 100% treatment success in children & 99% in adults. Usability research assessed GVOKE PFS and GVOKE HypoPen resulted in ~100% success rate in administering a full dose of glucagon utilizing 2-step administration process
• GVOKE is the first premixed, prefilled, premeasured liquid glucagon administered via prefilled syringe (GVOKE PFS) or auto-injector (GVOKE HypoPen) in patients with severe hypoglycemic events and will be available in 0.5 mg/0.1 mL dose and a 1 mg/0.2 mL dose for pediatric and adolescent/adult patients respectively

2. Ardelyx’s Ibsrela (tenapanor) Received the US FDA’s Approval for Irritable Bowel Syndrome with Constipation (ISB-C) for Adults

Published: Sept 12, 2019 | Tags: Ardelyx, Ibsrela, tenapanor, Receives, the US FDA, Approval, Irritable Bowel Syndrome, Constipation (ISB-C), Adults

• The approval was based on P-III IBS-C program that included Trial 1 (NCT02686138) and Trial 2 (NCT02621892) which involved assessing of Ibsrela vs PBO in patients with abdominal pain and bowel movement frequency
• The P-III IBS-C program met all 1EPs including Trial 1 and 2 (37%, 27% vs 24%, 19%), discontinuation rates (7.6% vs 0.8%)
• Ibsrela (50 mg, bid) is an orally administered drug involves inhibiting of sodium/hydrogen exchanger 3 (NHE3) targeted for the treatment of irritable bowel syndrome with constipation (IBS-C) in adults

3. Metacel Pharmaceuticals’ Ozobax (baclofen) Received the US FDA’s Approval for Spasticity Due to Multiple Sclerosis

Published: Sept 18 , 2019 | Tags: Metacel Pharmaceuticals, Ozobax, Baclofen, Receives, The Us Fda, Approval, Spasticity, Multiple Sclerosis

• Ozabax is a gamma-aminobutyric acid (GABA-ergic) agonist orally administered in low dosages and is available as a 5mg/5 ml oral solution
• The efficacy of Ozobax is based on the bioavailability study evaluated in healthy adults in comparison with Baclofen oral tablets
• Metacel is also planning to launch Ozobax with pending plans. Ozobax has a warning of for its withdrawal which can lead to neonatal withdrawal symptoms, drowsiness and sedation, poor tolerability in stroke patients, schizophrenia, or confusional states, and ovarian cysts

4. Novo Nordisk’s Rybelsus (semaglutide) Received FDA’s Approval as the First Oral GLP-1 Analog for Type 2 Diabetes

Published: Sept 23, 2019 | Tags: Approval, FDA, Glp-1, Analog, Novo Nordisk, Receives, Rybelsus, Semaglutide, Type-2 Diabetes

• The FDA’s approval was based on 10 PIONEER clinical studies assessing Rybelsus vs sitagliptin, empagliflozin, and liraglutide in 9,543 patients with T2D as an adjunct to diet & exercise
• The clinical studies resulted in A1C reduction as 1EPs & reduction in body weights as 2EPs. Additionally, in H1’19, Novo Nordisk acquired a tableting and packaging facility in Durham to meet anticipated supply needs for Rybelsus
• Rybelsus (qd) is an oral glucagon-like peptide-1 (GLP-1) analog, approved in two therapeutic doses 7/14mg with its expected availability in the US in Q4’19 and is under EMA & PMDA’s review. The US FDA is also reviewing Ryblesus for an additional indication, reducing the risk of MACE with an expected decision in Q1’20

5. Galderma’s Aklief (trifarotene) Received the US FDA’s Approval for the Treatment of Acne in 20 Years

Published: Oct 04, 2019 |Tags: Galderma, Aklief, trifarotene, Receives, the US FDA, Approval, Treatment, Acne,20 Years

• The approval was based on two P-III study results assessing Aklief (trifarotene, qd) vs vehicle in 2,420 patients with moderate acne on the face and trunk
• The P-III studies demonstrated reduction of inflammatory lesions as early as two weeks on the face and four weeks on the back, shoulders, and chest compared to vehicle with well-tolerated results
• Aklief (trifarotene) Cream 0.005% is a topical treatment used for targeting retinoic acid receptor (RAR) gamma designed for the treatment of acne vulgaris in patients 9 years of age and older with its expected availability in Nov 2019

6. Alvogen and Pfenex’s PF708 (biosimilar, teriparatide) Received FDA’s Approval for the Treatment of Osteoporosis

Published: Oct 07, 2019 | Tags: Alvogen, Pfenex, PF708, biosimilar, teriparatide, Receives, FDA, Approval, Treatment, Osteoporosis

• The US FDA granted approval to PF708, a biosimilar of Eli Lilly’s Forteo (teriparatide injection) under the 505(b)(2) regulatory pathway for the treatment of osteoporosis in certain patients at high risk for fracture
• Pfenex received a $2.5M milestone from Alvogen on its approval in the US. The PF708 is the first approved product of Pfenex, validating Pfenex Expression Technology platform and enhances patient’s access to a cost-effective alternative to Forteo
• Pfenex is conducting a comparative human factors study between PF708 and Forteo, evaluating the therapeutic equivalence of both the therapies with its anticipated submission by the end of Oct’19

7. Clinuvel’s Scenesse Received the US FDA’s Approval for Erythropoietic Protoporphyria (EPP)

Published: Oct 08, 2019 | Tags: Approval, Clinuvel, Erythropoietic Protoporphyria, Receives, Scenesse, the US FDA

• Scenesse (afamelanotide 16mg) is a drug developed for the treatment of rare genetic metabolic disorder and is approved to increase pain-free light exposure in adult patients with a history of phototoxic reactions from erythropoietic protoporphyria (EPP)
• In 2018, Scenesse also received European Medicines Agency’s approval as an orphan medicinal product, for EPP and is administered as a 16mg controlled-release injectable implant, designed for up to 60 days
• Scenesse (afamelanotide, 16mg) is a drug targeted for binding to melanocortin-1 receptor on skin cells and the results of P-III study has been published in the New England Journal of Medicine

8. Novartis’ Beovu (brolucizumab) Received FDA’s Approval for the Treatment of Wet Age-Related Macular Degeneration

Published: Oct 08, 2019 | Tags: Approval, Beovu, Brolucizumab, FDA, Novartis, Receives, Treatment, Wet, Age-Related, Macular Degeneration

• The approval was based on P-III HAWK (NCT02307682) & HARRIER (NCT02434328) studies that involved assessing of Beovu (intravitreal injection), 6mg (HAWK and HARRIER) & 3mg (HAWK) vs Aflibercept (2mg) in 1,800+ patients with wet AMD across 400 centers globally
• The P-III studies demonstrated non-inferiority in mean change BCVA @1yr. (week 48); reductions in central subfield thickness, @1yr. 56% & 51% maintained on the 3mos. dosing interval
• Beovu (RTH258) is the only anti-VEGF in wet AMD recommended to maintain eligible patients on up to 3mos. dosing intervals immediately after loading phase with no compromise in efficacy

9. Foamix’s Amzeeq (Topical Minocycline) Received the US FDA’s Approval for Moderate to Severe Acne

Published: Oct 18, 2019 | Tags: Amzeeq, Approval, FDA, Foamix, Moderate to Severe, Acne, Receives, Topical Minocycline, US

• The approval was based on P-III clinical studies evaluating Amzeeq (topical foam, 4%) versus vehicle in 2,418 patients aged ≥9yrs. with inflammatory lesions of non-nodular moderate to severe acne vulgaris
• The P-III studies resulted in improvement by the reduction of inflammatory lesions and improvement in IGA treatment success defined as score of 0 (“clear”) or 1 (“almost clear”) and at least a two-point decrease from baseline, well-tolerated with no AEs
• Amzeeq (FMX101) is the first topical minocycline to be approved by the FDA for any condition and is indicated to treat inflammatory lesions of non-nodular moderate to severe acne vulgaris in adults and pediatric patients with its anticipated availability in the US in Jan’2020

10. Vertex’s Trikafta (elexacaftor/tezacaftor/ivacaftor and ivacaftor) Received the US FDA’s Approval to Treat the Underlying Cause of Cystic Fibrosis

Published: Oct 22, 2019 | Tags: Ivacaftor, Approval, Cystic Fibrosis, Elexacaftor, FDA, Receives, Tezacaftor, Treat, Trikafta, US Vertex

• The approval was based on two global P-III studies assessing Trikafta in 403 & 107 patients with CF aged ≥12yrs. with at least one F508del mutation and one minimal function mutation (F/MF) & in people with two F508del mutations (F/F) for 24wks. & 4wks. respectively
• The two P-II studies result demonstrated improvement in FEV1 and respiratory symptoms in F/MF study @24wks. with a reduction in the rate of pulmonary exacerbations and improvements in BMI
• Trikafta is a prescription therapy to treat CF, currently being evaluated in P-III study in patients aged 6-11yrs with F/MF and F/F CF mutations and will be evaluated in children aged <6yrs. Vertex has submitted EMA’s MAA for the combination regimen 

11. Biogen and Alkermes’ Vumerity (diroximel fumarate) Received FDA’s Approval for Multiple Sclerosis

Published: Oct 30, 2019 | Tags: Regulatory, Taggedalkermes, Approval, Biogen, Diroximel Fumarate, FDA, Multiple Sclerosis, Receive, Vumerity

• The FDA’s approval was based on NDA submitted under the 505(b)(2) filing pathway and contain data from P-III EVOLVE-MS-1 study assessing Vumerity in patients with relapsing-remitting MS
• In Nov’2017, Biogen and Alkermes collaborated to develop & commercialize Vumerity under which Alkermes received $150M and royalties on WW sales of Vumerity for the 5yrs. following the US FDA’s approval
• VUMERITY is novel fumarate referencing Tecfidera (dimethyl fumarate) with a distinct chemical structure and is approved in the US to treat relapsing MS, being evaluated for improved patient-reported gastrointestinal tolerability