Insights+: The US FDA New Drug Approvals in August 2019

 Insights+: The US FDA New Drug Approvals in August 2019

Novel Drug Approvals by the US FDA in 2018

The US FDA’s Center for Drug Evaluation and Research (CDER) and Center for Biologics Evaluation and Research (CBER) have approved 31 new products so far in 2019. In 2018 FDA approved 59 novel products including 42 New Chemical Entity and 17 Biologics while breaking its past year’s records of approval. 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 8 new drugs approved by the US FDA in August 2019.

1.Daiichi Sankyo’s Tugaloo (pexidartinib) Received the US FDA’s Approval for the Symptomatic Tenosynovial Giant Cell Tumor

Published: Aug 02, 2019 | Tags: Approval, Daiichi Sankyo, The US FDA, Pexidartinib, Received, Symptomatic Tenosynovial Giant Cell Tumor, Turalio, The US

The FDA’s approval was based on P-III ENLIVEN study results assessing Turalio vs PBO in 120 patients in the ratio (1:1) with symptomatic TGCT associated with severe morbidity or functional limitations and not amenable to improvement with the surgery. The P-III ENLIVEN study results: @25wks. tumor response rate by RECIST v 1.1 (38% vs 0%); ORR by TVS (56% vs 0%). Turalio is approved with a boxed warning for hepatotoxicity due to the risk of liver injury. Turalio is the first oral therapy targeting CSF1R thus inhibiting the driver of abnormal cells in the synovium responsible for causing TGCT and has received FDA’s PR, BT, OD designation, currently under EMA’s review for TGCT.

2. TB Alliance’s Pretomanid Combination Therapy Received the US FDA’s Approval for the Treatment for Highly Drug-Resistant Forms of Tuberculosis  

Published: Aug 14, 2019 | Tags: Approval, Combination, Drug-Resistant, Forms, Highly, Pretomanid, Received, Tb Alliance, The US FDA, Therapy, Treatment, Tuberculosis

The approval was based on the Nix-TB trial assessing bedaquiline, pretomanid and linezolid collectively called BPaL regimen in 109 patients with XDR-TB treatment-intolerant or non-responsive MDR-TB across three sites in South Africa. The study demonstrated successful outcomes in @6mos. 95/107 patients and for two of the patients, the study was extended to nine months. The NDA submission contains data of 1,168 people receiving pretomanid in 19 clinical trials, evaluating its safety and efficacy. Pretomanid (PO) is an anti-TB drug in combination with bedaquiline + linezolid, and has received Priority Review, Qualified Infectious Disease Product, and Orphan Drug Designation with expected availability at the end of H2’19. Additionally, the company also submitted to the EMA.

3. Roche’s Rozlytrek (entrectinib) Received the US FDA Approval for ROS1-Positive Metastatic Non-Small Cell Lung Cancer and NTRK Gene Fusion-Positive Solid Tumors

Published: Aug 15, 2019 | Tags: Approval, Entrectinib, FDA, Genentech, Metastatic, NSCLC, NTRK, Gene Fusion-Positive Solid Tumors, Received, Ros1-Positive, Rozlytrek, US

The US FDA granted approval to Rozlytrek for ROS1-positive m-NSCLC in adults and accelerated approval for solid tumours having NTRK gene fusion in adults & pediatric patients aged ≥ 12yrs. The approvals were based on integrated analysis of P-II STARTRK-2, P-I STARTRK-1, P-I ALKA-372-001 and P-I/II STARTRK-NG study and resulted in 78% ORR, 1.8-36.8+ mos. DoR in ROS1-positive, m-NSCLC patients and 57% ORR and 2.8 to 26.0+ mos. DoR in patients with NTRK gene fusion-positive, LA/m-solid tumours. Rozlytrek (PO) is the first selective tyrosine kinase inhibitor targeting TRK A/B/C and ROS1 proteins, blocking ROS1 and NTRK kinase activity thus resulting in the death of cancer cells with ROS1 or NTRK gene fusions.

4. Harmony Biosciences’ Wakix (pitolisant) Received the US FDA’s Approval for Treatment of Excessive Daytime Sleepiness in Adult Patients with Narcolepsy

Published: Aug 15, 2019 | Tags:  Adult, Approval, Daytime, Excessive, Harmony, Biosciences, Pitolisant, Received, Sleepiness, The US FDA, Treatment, Wakix, Narcolepsy

The US FDA approval was based on HARMONY 1 and HARMONY 1b clinical study results assessing Wakix (pitolisant) vs PBO or active control in 261 narcolepsy patients. The study resulted in significant improvement in EDS as measured by the Epworth Sleepiness Scale (ESS) score. Wakix (pitolisant) is a selective histamine3 (H₃) receptor antagonist/inverse agonist will be available in Q4’19 and has also received Orphan Drug Designation for the treatment of narcolepsy in 2010. In 2017, Harmony in-licensed Pitolisant from Bioprojet.

5. AbbVie’s Rinvoq (upadacitinib) Received the US FDA’s Approval for Moderate to Severe Rheumatoid Arthritis

Published: Aug 16, 2019 | Tags: AbbVie, Approval, FDA, Moderate, Receives, Rheumatoid Arthritis, Rinvoq, Severe Upadacitinib, The US

The US FDA’s approval was based on P-III SELECT program including 5 studies results assessing Rinvoq (15mg) (SELECT-EARLY &-MONOTHERAPY, Rinvoq vs MTX) (SELECT-COMPARE, Rinvoq + MTX vs PBO + MTX) and (SELECT NEXT & -BEYOND, Rinvoq + csDMARDs vs PBO+ csDMARDs) in ~ 4,400 patients with mod. to sev. active RA who have had an inadequate response or intolerance to methotrexate (MTX-IR). The P-III five SELECT studies result: met its 1EPs & 2EPs i.e, @12wks. MTX-naive patients achieved ACR50 (52% vs 28%); @14wks. MTX-IR patients achieved ACR20 (68% vs 41%); @12wks. MTX-IR patients achieved ACR20 (71% vs 36%); csDMARD-IR patients achieved ACR20 (64% vs 36%); biologic-IR patients achieved ACR20 (65% vs 28%); inhibition in radiographic progression even without MTX. Rinvoq is an oral JAK inhibitor being studied for mod. to sev. RA and other immune-mediated diseases. It is under regulatory review in EU, Canada & Japan with its expected availability in the US in late Aug’2019.

6. Celgene’s Inrebic (fedratinib) Received the US FDA’s Approval for Myelofibrosis   

Published: Aug 19, 2019 | Tags: Approval, Celgene, FDA, Fedratinib, Inrebic, Myelofibrosis, Received, The US

The approval was based on multiple studies including JAKARTA and JAKARTA2. The P-III JAKARTA study involves assessing of Inrebic (400mg) vs PBO in patients with intermediate-2 or high-risk, primary or secondary (post-polycythemia vera or post-essential thrombocythemia) myelofibrosis with splenomegaly and a platelet count of ≥50 x 109/L2 prior not treated with JAK inhibitor. The P-III JAKARTA study results: @24wks. with 4wks. follow up scan, >35% reduction in spleen volume (37% vs 1%); >50% improvement in TSS as measured by (MFSAF) v2.0 diary (40% vs 9%). Inrebic is an oral kinase inhibitor targeting JAK2 & FLT3 is a new therapy in nearly a decade for patients affected by rare bone marrow cancer and is gained by Celgene with the acquisition of Impact Biomedicines for $7B. Inrebic also has a Boxed Warning of serious and fatal encephalopathy, including Wernicke’s.

7. Nabriva’s Xenleta (lefamulin) Received FDA’s Approval for the Treatment of Community-Acquired Bacterial Pneumonia in Adults 

Published: Aug 19, 2019 | Tags: Approval, Community-Acquired Bacterial Pneumonia, FDA, Lefamulin, Nabriva, Received, Treatment, Xenleta

The US FDA’s approval was based on two P-III studies, LEAP 1 & LEAP 2. LEAP 1 involves assessing Xenleta (150mg IV/600mg oral for 5-7days) vs moxifloxacin (IV/oral for 7days) with/out linezolid in patients with CABP which demonstrated non-inferiority to moxifloxacin i.e, ECR rate (87.3% vs 90.2%). The P-III LEAP 2 study involves assessing Xenalta (PO for 5days) vs moxifloxacin (PO for 7days) which resulted in achieving similar ECR rates in IIT population. Xenleta (lefamulin) is a semi-synthetic pleuromutilin antibiotic positioned for use as a 1L therapy for CABP, targeting in vitro spectrum of activity against Gram+ and Gram- pathogens associated CABP and has received FDA’s QIDP & FT designation with its expected availability in mid-Sept’2019.

8.Kyowa Kirin’s Nourianz (istradefylline) Received FDA’s Approval as an Adjunctive Therapy in Patients with Parkinson’s Disease 

Published: Aug 28, 2019 | Tags: Adjunctive Therapy, Approval, FDA, Istradefylline, Kyowa Kirin, Nourianz, Parkinson’s Disease, Patients, Received

The US FDA has granted approval to Nourianz for use as an add-on treatment to levodopa/carbidopa which is based on the clinical studies assessing Nourianz vs PBO in PD patients experiencing “OFF” episodes, administering a stable dose of levodopa/carbidopa with/out other PD therapies. The four clinical study results demonstrated that in adjunct to levodopa/carbidopa, istradefylline improves OFF time and is well tolerated among the patients. Nourianz (PO) is a selective adenosine A2A receptor antagonist and is marketed under the trade name as Nouriast in Japan indicated for the improvement of the wearing-off phenomenon in patients with PD, since May 30, 2013.


Vartika Singh

Vartika Singh is a content writer who loves to write research articles and reports at PharmaShots. She has in-depth knowledge of the life sciences industry including the Pharma and Biotech sectors. Any articles written by her can be contacted at

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