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

The US FDA has approved multiple NDAs and BLAs in May 2020, leading to the 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 43 novel products so far in 2020, including 12 in May 2020
Additionally, last year in 2019, the US FDA has approved 48 novel products. We have compiled a list of a total of 12 new drugs approved by the US FDA in May 2020.

1. Dr. Reddy's Elyxyb (celecoxib oral solution) Received the US FDA's Approval for Acute Treatment of Migraine with or Without Aura in Adults

Published: May 01, 2020 | Tags: Dr. Reddy, Elyxyb, celecoxib oral solution, Received, the US FDA, Approval, Acute, Treatment, Migraine, Without, Aura, with, Adults

Elyxyb (previously as DFN-15) is an oral solution formulated using a self-micro emulsifying drug delivery system helps in improving solubility and bioavailability of the drug leading for absorption
The candidate demonstrated a rapid onset of actions important for suffering from acute migraine and resulted in efficacy with few AEs
NSAIDs including ELYXYB can lead to an increase in the risk of bleeding events also co-morbid conditions such as coagulation disorders

2. Genmab and Janssen's Darzalex Faspro (daratumumab and hyaluronidase-fihj) Received the US FDA's Approval to Treat Multiple Myeloma in Adults

Published: May 01, 2020 | Tags: Genmab, Janssen, Darzalex Faspro daratumumab, hyaluronidase-fihj, Received, the US FDA, Approval, Treat, Multiple Myeloma, Adults

The approval is based on two studies: COLUMBA (MMY3012, P-III) study and P-II PLEIADES (MMY2040) study, which involves assessing of subcutaneous daratumumab (1800 mg) with rHuPH20 2,000 U/mL, combination with either bortezomib, lenalidomide and dexamethasone (D-VRd) or bortezomib, melphalan and prednisone (D-VMP)
In Feb 2019, results from COLUMBA study were announced, presented in oral sessions at the 2019 ASCO Annual Meeting and the 24th European Hematology Association (EHA) Annual Congress. Additionally, in Dec 2019, an update of the COLUMBA data as well as data from the PLEIADES study was presented during poster sessions at the 61st ASH Annual Meeting
DARZALEX FASPRO is a fixed-dose formulation administered every three to five minutes to patients with multiple myeloma in the US

3. Tolmar's Fensolvi Received the US FDA's Approval to Treat Pediatric Patients Two Years of Age and Older with Central Precocious Puberty (CPP)

Published: May 04, 2020 | Tags: Tolmar, Fensolvi, Received, the US FDA, Approval, Treat, Pediatric Patients, Two, Years, Age, Older, Central Precocious Puberty, CPP

The approval was based on P-III study which involves assessing of leuprolide acetate (LA,45 mg) in 64 children with central (gonadotropin-dependent) precocious puberty evaluating its safety and pharmacokinetics
The study achieved its 1E i.e. 87% of children achieved a serum luteinizing hormone concentration of <4 IU/L at six months post-injection with most common TEAEs were injection site pain (31%), nasopharyngitis (22%), and fever (17%)
Fensolvi (leuprolide acetate) is a novel six-month, subcutaneous leuprolide acetate gonadotropin-releasing hormone (GnRH) agonist with small injectable suspension approved for the treatment of pediatric patients two years of age and older with Central Precocious Puberty

4. Novartis and Incyte's Tabrecta (capmatinib, INC280) Received the US FDA's Approval for Metastatic Non-Small Cell Lung Cancer with METex14

Published: May 07, 2020 | Tags: Approval, Capmatinib, Inc280, FDA, Incyte, Metastatic, Non-Small Cell Lung Cancer, Metex14, Novartis, Received, Tabrecta, US

The approval was based on the P-II GEOMETRY mono-1 study assessing Tabrecta (400mg, bid) in 97 patients with metastatic NSCLC harbouring mutations that lead to METex14. The US FDA approved the therapy under accelerated approval based on ORR & DOR
The P-II study results: In treatment naïve & previously treated patients, ORR (68% & 41%); DOR (12.6 & 9.7mos.) respectively. Additionally, the FDA has approved FoundationOne CDx as the CDx for Tabrecta, to help in detecting mutations leading to MET exon 14 skipping in tumor tissue
Tabrecta (capmatinib) is a kinase inhibitor targeting MET, received the US FDA's BT designation and is expected to be available to patients in the coming days. In 2009, Incyte granted Novartis WW exclusive development and commercialization rights to Tabrects and certain back-up compounds in all indications

5. Eli Lilly's Retevmo (selpercatinib) Received the US FDA's Approval as the First Therapy to Treat Advanced RET-Driven Lung and Thyroid Cancers

Published: May 11, 2020 | Tags: Advanced, Ret-Driven, Lung, Approval, Eli Lilly, First Therapy, Received, Retevmo, Selpercatinib, The US FDA, Thyroid, Cancers, Treat

Approval was based on LIBRETTO-001 P-I/II study, which is assessing Retevmo (selpercatinib, 40 mg & 80 mg capsules) in 702 patients with both treatment-naive and heavily pre-treated solid tumor patients (incl. RET fusion-positive NSCLC, RET-mutant MTC, RET fusion-positive thyroid cancer, and other solid tumors with RET alterations)
The P-I/II study results (treatment naïve; treatment experienced): RET Fusion-Positive NSCLC (N=39; 105), ORR (85%; 64%), mDOR (NR vs 17.5 mos.); RET-mutant MTC (N=88; 55) ORR (73%; 69%), mDOR (22 mos.; NR), RET Fusion-Positive Thyroid Cancers (N= 8, 19), ORR (100%; 79%), mDOR (NR; 7.6 mos.)
Retevmo (formerly LOXO-292) is an oral selective RET kinase inhibitor (120/160mg by -/+50kg weight) BID, and has also received the FDA ODD for RET fusion +ve NSCLC, RET fusion +ve & RET-mutant thyroid cancers, undifferentiated or anaplastic thyroid cancer, MTC and LA or metastatic follicular or papillary thyroid cancer

6. Deciphera Pharmaceuticals Qinlock (ripretinib) Received the US FDA's Approval to Treat 4L Gastrointestinal Stromal Tumor

Published: May 15, 2020 | Tags: 4L, Approval, Deciphera Pharmaceuticals, Gastrointestinal Stromal Tumor, Qinlock, Received, Ripretinib, The US FDA, Treat

The approval was based on P-III INVICTUS study including combined safety results from the P-I study which assessing of Qinlock (150mg, qd) vs PBO in a ratio (2:1) in patients with advanced GIST whose previous therapies have included imatinib, sunitinib, and regorafenib to evaluate its safety, tolerability, and efficacy
The study demonstrated median PFS (6.3 mos. vs 1.0 mos.), reduced the risk of disease progression or death by 85%, mORR of 9.4%, mOS (15.1 mos. vs 6.6 mos.), reduced the risk of death by 64%
Qinlock (ripretinib) is a kinase inhibitor targeted to treat adult patients with advanced gastrointestinal stromal tumor (GIST)

7. Zionexa And Petnet Solutions Cerianna (Fluoroestradiol F 18) Received the US FDA's Approval for Advancing Treatments in Metastatic Breast Cancer Patients

Published: May 15, 2020 | Tags: Zionexa, Petnet Solutions, Cerianna, Fluoroestradiol F 18, Received, the US FDA, Approval, Advancing, Treatments, Metastatic, Breast Cancer, Patients

Cerianna (Fluoroestradiol F 18) is a novel FDA approved molecular imaging agent targeted for PET imaging for detection of estrogen receptor-positive lesions as an adjunct to biopsy in patients with recurrent or metastatic breast cancer
The product will also allow the clinicians better guide treatments for metastatic breast cancer patients and improve patients QoL
In late 2020/early 2021, the PETNET Solutions will be responsible for Cerianna (Fluoroestradiol F 18) commercialization while Zionexa will manufacturer and exclusive commercialize it in the US

8. Amivas's Artesunate Received the US FDA's Approval for Treatment of Severe Malaria

Published: May 15, 2020 | Tags: Amivas, Artesunate, Received, the US FDA, Approval, Treatment, Severe Malaria

Amivas Artesunate for Injection (110 mg) is a powder and solvent for solution US FDA approved and is targeted for initial treatment of severe malaria in adult and pediatric patients
Artesunate's (IV) safety and efficacy was examined vs standard of care drug in three trials including the South East Asian Quinine Artesunate Malaria Trial (SEAQUAMAT) and the African Quinine Artesunate Malaria Trial (AQUAMAT) enrolling 6,886 patients and reported reduced mortality (34.7% vs 22.5%)
The CDC will continue the process of manufacturing investigational IV artesunate available under the CDC-sponsored expanded access IND 76,725, in effect per FDA's authorization since 2007 until nationwide availability of Amivas-produced Artesunate for Injection is in place in the next few months

9. Sunovion Pharmaceutical's Kynmobi (apomorphine hydrochloride) Received the US FDA's Approval to Treat Parkinson's Disease Off Episodes

Published: May 21, 2020 | Tags: Sunovion Pharmaceutical, Kynmobi, apomorphine hydrochloride, Receives, US FDA, Approval, Treat, Parkinson Disease, off, Episodes

The approval is based on P-III study which involved assessing of Kynmobi vs PBO in patients with Parkinson's Disease Off Episodes
The study resulted in improvements in motor symptoms at 30 minutes after dosing @12 wks., the mean reduction of 7.6 points, initial clinical improvements were seen at 15 minutes post-administration full ON response within 30 minutes @12wks., well-tolerated
Kynmobi sublingual film used to treat short-term (acute), intermittent 'off' episodes in people with Parkinson's disease with expected availability in Sep 2020

10. Astellas VESIcare LS (solifenacin succinate) Received the US FDA's Approval to Treat Neurogenic Detrusor Overactivity (NDO) In Pediatric Patients Aged 2 Years and Older

Published: May 27, 2020 | Tags: Astellas, VESIcare LS, solifenacin succinate, Received, the US FDA, Approval, Treat, Neurogenic Detrusor Overactivity, NDO, Pediatric, Patients, Aged, 2 Years, Older

VESIcare LS is an oral suspension dosage prescribed medicine specifically developed to facilitate dosing and administration for children 2 years of age and older with a condition called neurogenic detrusor overactivity
In 2004, the VESIcare LS initially approved in the US while its oral suspension will come in a 5 mg/5 mL (1 mg/mL) oral suspension and will be available in the US in late 2020
The product is usually used for the reduction of urine your bladder can hold and reduce urine leakage

11. Eli Lilly's Tauvid (flortaucipir F 18) Received the US FDA's Approval as the First Drug to Image Tau Pathology in Patients Being Evaluated for Alzheimer's Disease

Published: May 29, 2020 | Tags: Eli Lilly, Tauvid, (flortaucipir F 18), Receives, US, FDA, Approval, First Drug, Image, Tau Pathology, Patients, Being, Evaluated, Alzheimer's Disease

The safety and effectiveness of Tauvid imaging was evaluated in two clinical studies. In study 1, reader interpretations of premortem TAUVID scans from 64 cognitively normal & terminally ill patients agreed to undergo Tauvid imaging and participate in a post-mortem brain donation program were compared to tau pathology at autopsy. The study met its 1EPs as with reader sensitivity ranging from 92%-100% & specificity from 52%- 92%
In study 2, images from the same terminally ill patients as in Study 1 and 159 patients with cognitive impairment being evaluated for AD were evaluated by 5 new readers. The study met criteria for comparison of Tauvid reads to NFT pathology, additionally, an inter-reader agreement was evaluated using Fleiss kappa statistic and found to be 0.87 across 241 patients in Study 2
The availability of Tauvid will initially be limited and will expand in response to commercial demand and payor reimbursement. Tauvid is the first and only approved diagnostic agent to image tau NFTs in the brain

12. AbbVie and Neurocrine's Oriahnn Receive the US FDA's Approval as the First Oral Therapy for the Management of Heavy Menstrual Bleeding Due to Uterine Fibroids in Pre-menopausal Women

Published: May 29, 2020 | Tags: AbbVie, Neurocrine, Oriahnn, Receive, the US FDA, Approval, First, Oral, Therapy, Management, Heavy, Menstrual Bleeding, Uterine Fibroids, Pre-menopausal Women

The approval follows P-III ELARIS UF-I and ELARIS UF-II studies assessing Oriahnn (elagolix, estradiol, and norethindrone acetate capsules; elagolix capsules) vs PBO for the management of heavy menstrual bleeding associated with uterine fibroids in premenopausal women for up to 24mos.
The study met its 1EPs of clinically meaningful reduction in bleeding (defined as the proportion of women who achieved both at least a 50% reduction in menstrual blood loss at the final month of treatment and a total menstrual blood loss amount of < 80 ml), 7/ 10 vs 1/10 women no longer experiencing heavy menstrual bleeding, reduction in bleeding due to uterine fibroids by 50% within the first month of use
The approval provides women with a non-surgical option addressing unresolved heavy menstrual bleeding. The therapy is expected to be available in the US by the end of Jun'2020