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

The US FDA has approved multiple NDAs and BLAs in July 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 59 novel products so far in 2020, including 8 in July 2020. Additionally, last year in 2019, the US FDA has approved 48 novel products. We have compiled a list of a total of 8 new drugs approved by the US FDA in July 2020.

1.Acacia Pharma’s Byfavo (remimazolam) Received the US FDA’s Approval for the Induction and Maintenance of Procedural Sedation

Published: Jul 02, 2020 | Tags: Acacia Pharma, Byfavo, remimazolam, Received, the US FDA, Approval, Induction, Maintenance, Procedural Sedation

• The safety of Byfavo was evaluated in 969 patients assessing Byfavo for the induction and maintenance of procedural sedation
• In Jan 2020, the company in-licensed the commercial rights to Byfavo and will pay $33.6M upfront generated by the approval, consisting of $16.8M in cash and $16.8M in ordinary shares of Acacia Pharma stock. Additionally, Acacia will get $11.2M net cash to launch the product in the US
• Byfavo (remimazolam) is an injection rapid onset/offset IV benzodiazepine sedative for the induction and maintenance of procedural sedation in adults undergoing procedures lasting 30 minutes or less, with its expected launch in the US in H2’20

2. Osmotica’s Upneeq Received the US FDA’s Approval to Treat Blepharoptosis (Droopy Eyelid) in Adults

Published: Jul 09, 2020 | Tags: Osmotica, Upneeq, Received, the US FDA, Approval, Treat, Blepharoptosis, Droopy Eyelid, Adults

• The approval was based on P-III trial assessing Upneeq vs PBO in a (2:1) ratio in 140 patients with acquired blepharoptosis split into two treatment groups for 42 days. Additionally, another second P-III clinical trial evaluated the safety and efficacy of Upneeq vs PBO in 35 patients to treat acquired blepharoptosis
• The study resulted in well-tolerated results when administered once daily over a six-week period with no serious AEs and met its both 1EPs and 2EPs, mean change from baseline on the LPFT on Hour 6, Day 1 6.3 vs 2.1, Hour 2 Day 14 7.7 vs 2.4, improvement in MRD-1 at 5 and 15 minutes, and 2 and 6 hours post-dose on days 1 and 14
• Upneeq (oxymetazoline hydrochloride ophthalmic solution) is a QD ophthalmic formulation of oxymetazoline and an alpha-adrenergic receptor agonist a novel pharmacologic treatment indicated for the treatment of acquired blepharoptosis

3. Otsuka Inqovi (decitabine and cedazuridine) Received the US FDA’s Approval to MDS and CMML

Published: Jul 20, 2020 | Tags: Otsuka, Inqovi, decitabine, cedazuridine, Received, the US FDA, Approval, MDS, CMML

• The approval was based on P-III trial from the ASCERTAIN study supporting P-I and P-II clinical studies which evaluated the safety and efficacy including decitabine exposure equivalence in oral Inqovi vs intravenous decitabine
• Otsuka a subsidiary of Astex Pharmaceuticals with its partner Taiho Pharmaceutical and North American reported the approval
• Inqovi is a novel product orally administered hypomethylating agent approved by the US and Health Canada for the treatment of MDS and CMML

4. Jazz Pharmaceuticals’ Xywav (calcium, magnesium, potassium, and sodium oxybates) Received the US FDA’s Approval for Cataplexy or Excessive Daytime Sleepiness Associated with Narcolepsy

Published: Jul 22, 2020 | Tags: Jazz Pharmaceuticals, Xywav, Calcium, magnesium, potassium, sodium oxybates, Received, the US FDA, Approval, Cataplexy, Excessive, Daytime, Sleepiness, Associated, Narcolepsy

• The FDA approval was based on P-III study results assessing Xywav vs PBO in 201 patients to evaluate the safety and efficacy in the treatment of cataplexy and EDS in patients with narcolepsy 
• The study resulted in statistically significant differences in the weekly number of cataplexy attack and Epworth Sleepiness Scale scores
• Xywav, also known as JZP-258 is targeted for the treatment of cataplexy or excessive daytime sleepiness in patients 7 years of age and older with narcolepsy. Additionally, the USDEA (Drug Enforcement Administration) has designated Xywav as a Schedule III medicine also contains a boxed warning of as a central nervous system depressant

5. MC2 Therapeutics Wynzora Cream Received the US FDA’s Approval to Treat Plaque Psoriasis in Adults

Published: Jul 22, 2020 | Tags: MC2 Therapeutics, Wynzora Cream, Received, the US FDA, Approval, Treat, Plaque Psoriasis, Adults

• The approval was based on P-III trial assessing Wynzora Cream vs Taclonex Topical Suspension in 794 patients for the treatment of plaque psoriasis in adults 18 years of age or older
• The study resulted in a PGA treatment of 21.6% vs 14.6%, patients achieved at least a 4-point improvement in the peak pruritus NRS score at @4wks. 60.3% vs 21.4%
• Wynzora is a novel topical solution offering high efficacy, favorable safety and excellent and is filled for MAA application in EU

6. AstraZeneca’s Breztri Aerosphere Received the US FDA’s Approval for the Maintenance Treatment of COPD

Published: Jul 24, 2020 | Tags: AstraZeneca, Breztri Aerosphere, Received, US, FDA, Approval, Maintenance Treatment, COPD

• The approval was based on P-III ETHOS study assessing of Breztri Aerosphere (budesonide/glycopyrronium/formoterol fumarate) vs dual therapies [Bevespi Aerosphere (glycopyrronium/formoterol fumarate) and PT009 (budesonide/formoterol fumarate)] with moderate to severe COPD and a history of exacerbation(s) in the previous year
• The P-III ETHOS study demonstrated a reduction in the rate of moderate or severe exacerbations. Additionally, the P-III KRONOS study also support the approval of the therapy
• Breztri Aerosphere is an approved therapy in Japan and China for patients with COPD and is under regulatory review in the EU. As per the agreement to acquire Pearl Therapeutics, AstraZeneca paid $150M as a milestone on US approval of the therapy for COPD

7. MorphoSys’ Monjuvi (tafasitamab-cxix) + Lenalidomide Received the US FDA’s Approval as 2L Treatment for Patients with Relapsed or Refractory Diffuse Large B-cell Lymphoma

Published: Jul 31, 2020 | Tags: MorphoSys, Monjuvi, tafasitamab-cxix, Lenalidomide, Received, US, FDA, Approval, 2L, Treatment, Patients, Relapsed, Refractory, Diffuse Large B-cell Lymphoma

• The approval was based on P-II L-MIND study assessing Monjuvi in combination with lenalidomide as a treatment for adult patients with relapsed or refractory DLBCL
• Results: ORR (55%); CR (37%); PR (18%); mDOR (21.7mos.). Additionally, the US FDA has granted BT designation for the combination therapy for r/r DLBCL
• Monjuvi is a humanized Fc-modified cytolytic CD19 targeting mAb, has been approved under US FDA’s accelerated approval, and is expected to be commercially available in the US. MorphoSys and Incyte will co-commercialize Monjuvi in the US & has exclusive commercialization rights outside the territory

8. ViiV Healthcare’s Rukobia (fostemsavir) Received the US FDA’s Approval for HIV in Patients with Limited Treatment Options

Published: Jul 31, 2020 | Tags:  ViiV Healthcare, Rukobia, fostemsavir, Received, US, FDA, Approval, HIV, Patients, Limited Treatment Options

• The approval was based on P-III BRIGHTE study assessing Rukobia (600mg, ER) + OBT in 371 HTE adults living with multidrug-resistant HIV. Participants were enrolled in either a randomized or nonrandomized cohort
• In the randomized cohort, 60% adults achieved undetectable HIV viral load and clinically meaningful improvements to CD4+ T-cell count @96wks., HIV-1 RNA <40 copies/mL @24 & 96wks. (53% & 60%); changes in CD4+ cell count (90 & 205 cells/mm3) respectively. In the nonrandomized cohort, 37% achieved HIV-1 RNA <40 copies/mL @24 & 96wks.; HIV-1 RNA <200 copies/mL (42% & 39%); mean changes in CD4+ cell count (41 & 119 cells/mm3) respectively
• Fostemsavir is a first-in-class HIV-1 attachment inhibitor, currently under EMA’s review with additional submissions to regulatory authorities anticipated in 2020 & 2021