Shots:    

• PharmaShots has compiled a list of US FDA-approved drugs in the month of August 2024    

• The US FDA has approved a total of 7 new drugs including 4 new molecular entities and 3 biologics leading to the treatments for patients and advances in the healthcare industry      

• The major highlighted drug was Johnson & Johnson’s Rybrevant plus Lazcluze for the treatment of EGFR mutant NSCLC 

1. Servier Reports the US FDA’s Approval of Voranigo (Vorasidenib) to Treat Grade 2 IDH-Mutant Glioma 

Product Name: Voranigo 

Active ingredient: Vorasidenib 

Company: Servier 

Date: Aug 06, 2024   

Disease: Grade 2 IDH-Mutant Glioma 

Shots:    

• The US FDA has granted approval to Voranigo (QD) tablets for treating patients (age: ≥12yrs.) with grade 2 astrocytoma or oligodendroglioma with a susceptible IDH1/2 mutation post-surgery 

• Approval was based on P-III (INDIGO) trial assessing the safety & efficacy of Voranigo vs PBO to treat residual or recurrent grade 2 glioma with IDH1/2 mutation after surgery 

• Study reached the 1EP of PFS & 2EP of TTNI with an mPFS of 27.7mos. vs 11.1mos. & median TTNI not attained vs 17.8mos.; tumor volume decreased by a mean of 2.5% with Voranigo & increased by a mean of 13.9% with PBO every 6mos. Data was published in the NEJM & highlighted at ASCO 2023 

2. The US FDA Approves Citius Pharmaceuticals’ Lymphir to Treat R/R Cutaneous T-Cell Lymphoma (CTCL) 

Product Name: Lymphir 

Active ingredient: Denileukin diftitox-cxdl 

Company: Citius Pharmaceuticals 

Date: Aug 07, 2024   

Disease: Cutaneous T-Cell Lymphoma (CTCL)  

Shots:   

• The US FDA has granted approval to Lymphir for treating chronic r/r CTCL in patients treated with at least one prior systemic therapy, with its introduction anticipated within the next 5mos. 

• The approval was based on P-III (Study 302) trial assessing the safety & efficacy of Lymphir (9mcg/kg, IV) among patients (n=69) with r/r CTCL (stage I to IV) 

• The study showed an ORR of 36.2%, CR in 8.7%, median time to response of 1.41mos. in ~70% post 1–2 cycles of treatment and DoR of at least 6mos. in 52%. 84.4% of them showed reduced skin tumor burden, with 12.5% achieving complete skin clearance. Pruritus improved in 31.7% of patients without any cumulative toxicity   

3. Ascendis Pharma’s Yorvipath Receives the US FDA’s Approval to Treat Hypoparathyroidism 

Product Name: Yorvipath    

Active ingredient: Palopegteriparatide   

Company: Ascendis Pharma 

Date: Aug 09, 2024    

Disease: Hypoparathyroidism 

Shots:     

• The US FDA has approved Yorvipath (QD) tablets for treating hypoparathyroidism patients, with its initial supply planned during Q1’25 

• The approval was supported by the P-II (PaTH Forward) as well as P-III (PaTHway) clinical trials conducted worldwide  

• Ascendis will further introduce the US Ascendis Signature Access Program (A.S.A.P.) for treatment support & financial assistance during the product launch. In addition, it seeks the US FDA’s approval to market currently manufactured product, will be launched by Q4’24 on approval 

4. Galderma Reports the US FDA’s Approval of Nemluvio (Nemolizumab) for Treating Prurigo Nodularis (PN) 

Product Name: Nemluvio  

Active ingredient: Nemolizumab 

Company: Galderma 

Date: Aug 12, 2024   

Disease: Prurigo Nodularis 

Shots:    

• The approval of Nemluvio (SC pre-filled pen) was based on P-III (OLYMPIA 1 & 2) studies assessing its safety & efficacy vs PBO in PN patients (n= >500) 

• Studies met their 1 & 2EPs, showing at least a 4-point reduced itch intensity in 56% & 49% vs 16% (both PBO groups); skin nodules clearance in 26% & 38% vs 7% & 11% and at least a 4-point reduced sleep disturbance in 50% & 52% vs 12% & 21% at wk.16. At least a 4-point reduced itch intensity in 41% (both studies) vs 6% & 7% was seen at wk.4 

• The US FDA is reviewing its BLA for moderate-to-severe AD, with a decision expected in H2’24; MAA in PN & AD are also under review in the EU, Canada, Australia, Singapore, Switzerland & the UK, with further submissions planned in 2024 

5. US FDA Grants Accelerated Approval to Gilead’s Livdelzi (Seladelpar) to Treat Primary Biliary Cholangitis (PBC) 

Product Name: Livdelzi 

Active ingredient: Seladelpar 

Company: Gilead 

Date: Aug 14, 2024   

Disease: Primary Biliary Cholangitis 

Shots:    

• Gilead’s Livdelzi (Seladelpar) as monotx. or in combination with ursodeoxycholic acid (UDCA) has received accelerated approval for adults unable to tolerate UDCA or with inadequate response to UDCA, respectively. Not recommended for individuals with decompensated cirrhosis 

• The approval was based on a P-III (RESPONSE) study showing 62% vs 20% (Livdelzi vs PBO) composite biochemical response at 12mos.; normalization of ALP value in 25% Livdelzi pts at 12mos. 

• For complete approval, confirmatory long-term study P-III (AFFIRM) has been initiated to evaluate Livdelzi's effect in compensated cirrhosis patients due to PBC. Seladelpar’s review has been accepted by MHRA and EMA 

6. Incyte and Syndax Receives US FDA Approval for Niktimvo (axatilimab-csfr) to Treat Chronic Graft-Versus-Host Disease (GvHD) 

Product Name: Niktimvo 

Active ingredient: Axatilimab-csfr 

Company: Incyte 

Date: Aug 14, 2024   

Disease: Graft-Versus-Host Disease 

Shots:    

• Based on P-II (AGAVE-201) study, Niktimvo got FDA approval to treat chronic GvHD patients who failed at least two lines of previous therapy 

• Niktimvo patients (0.3 mg/kg, Q2W, n=79) achieved 75% ORR in 6mos. with 1.5mos. mToR; 60% maintained response at 12mos. Study also met key exploratory EPs, 56% patients achieved ≥7-point improvement in mLSS score; 44% shown SAEs 

• Both companies will co-commercialize Niktimvo in US, whereas Incyte has exclusive commercial rights outside the US. Companies plan to seek approval for two smaller Niktimvo vials following the US FDA approval (50mg size), anticipated US launch in early Q1'25 

7. Johnson & Johnson Reports the US FDA’s Approval of Rybrevant + Lazcluze as a 1L Treatment of NSCLC with EGFR Mutations 

Product Name: Lazcluze 

Active ingredient: Lazertinib   

Company: Johnson & Johnson  

 Date: Aug 19, 2024   

 Disease: Non-Small Cell Lung Cancer 

 Shots:    

• The US FDA has approved J&J’s Rybrevant + Lazcluze as a 1L treatment for LA/metastatic NSCLC with EGFR exon 19 deletions or exon 21 L858R substitution mutations 

• The approval was based on the P-III (MARIPOSA) assessing Rybrevant + lazertinib vs osimertinib and lazertinib alone in 1074 patients 

• The study reached its 1EP and 2EPs vs osimertinib that shows mPFS (23.7mos. vs 16.6mos.) & 2EP shows mDOR (25.8mos. vs 16.7mos.). These data were presented at the ESMO 2023 & ASCO 2024 and published in the NEJM & Annals of Oncology 

Related Post: Insights+: The US FDA New Drug Approvals in July 2024