Shots:  

• PharmaShots has compiled a list of US FDA-approved drugs in the month of April 2024  
• The US FDA approved a total of 12 new drugs including 11 new molecular entities and 1 biologic leading to the treatments for patients and advances in the healthcare industry    
• The major highlighted drug was X4 Pharmaceuticals’ Xolremdi for the treatment of WHIM Syndrome  

1. Basilea’s Zevtera Receives the US FDA’s Approval for Staphylococcus aureus Bloodstream Infections 

               Active Ingredient: Ceftobiprole medocaril sodium 

               Approved:  Apr 03, 2024  

               Company: Basilea Pharmaceutica 

               Disease: Staphylococcus aureus bloodstream infections (bacteremia) incl.

                                    Right-sided infective endocarditis

                                    Acute bacterial skin and skin structure infections (ABSSSI)  

                                    Community-acquired bacterial pneumonia (CABP) 

• The US FDA has approved Zevtera to treat adult patients with SAB infections such as right-sided infective endocarditis, acute bacterial skin and skin structure infections (ABSSSI) as well as community-acquired bacterial pneumonia (CABP) in adult & pediatric patients (3mos. to <18yrs.)  

• The NDA was based on efficacy & safety results from P-III trials incl. ERADICATE (for SAB), TARGET (for ABSSSI) & a P-III trial for CABP 

• Additionally, Basilea was awarded ~$112M from US HHS, ASPR & BARDA for the P-III study of Zevtera, covering ~75% cost of SAB & ABSSSI studies, regulatory efforts & non-clinical work  

2. ImmunityBio’s Anktiva Receives the US FDA’s Approval to Treat Non-Muscle Invasive Bladder Cancer (NMIBC) 

               Active Ingredient: Nogapendekin alfa inbakicept-pmln 

               Approved: Apr 22, 2024  

               Company: ImmunityBio 

               Disease: Non-Muscle Invasive Bladder Cancer  

• ImmunityBio’s Anktiva + Bacillus Calmette-Guérin (BCG) has received the US FDA’s approval to treat BCG-unresponsive non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ (CIS) and with/without papillary tumors. The US availability is anticipated by mid-May 2024 

• The approval & BTD was based on the study of 77 evaluable patients depicting a CR rate 62% with the confidence interval of 73% and DoR >47mos, as of Nov 2023 

• The DoR & subsequent mDoR evaluation is in progress. Among 58% CR patients, DoR is ≥12mos. & 40% had ≥24mos. DoR 

3. The US FDA Grants Accelerated Approval to Day One’s Ojemda for Treating Pediatric Low-Grade Glioma (pLGG) 

               Active Ingredient: Tovorafenib  

               Approved: Apr 23, 2024  

               Company: Day One Biopharmaceuticals 

               Disease: Pediatric Low-Grade Glioma 

• The US FDA has granted accelerated approval to Ojemda for treating r/r pLGG patients (≥6mos.) with BRAF fusion/rearrangement or BRAF V600 mutation. It further obtained FDA’s rare pediatric disease priority review voucher  

• The approval was based on the P-II (FIREFLY-1) study assessing the efficacy (arm 1, n=77) & safety (arm 2, n=60) of Ojemda. Also enrolling patients for P-III (FIREFLY-2/LOGGIC) studies of tovorafenib as front-line therapy in pLGG patients (6mos. to 25yrs.) in US, Canada, EU, Australia & Asia 

• The results, in arm 1 (n=76 evaluable; data cutoff: Jun 5, 2023), showed 51% ORR, incl. 28% PRs & 11% MRs; 52% & 50% ORR in patients with BRAF fusions or rearrangements (n=64) & with BRAF V600 mutation (n=12), respectively; 49% & 55% ORR in patients with prior MAPK-targeted therapy (n=45) and without it (n=31), respectively; 13.8mos. mDoR, whereas mToR was 5.3mos. and ORR is 53% by RANO LGG criteria. 66% of patients remain on study 

4. Bausch + Lomb’s Lumify Receives the US FDA’s Approval to Treat Ocular Redness  

               Active Ingredient: Brimonidine tartrate 

               Approved: Apr 19, 2024  

               Company: Bausch + Lomb Corporation                  

               Disease: Ocular Redness  

• Bausch + Lomb has received the US FDA’s approval for Lumify Preservative Free eye drops to treat ocular redness due to minor eye irritations 

• The studies demonstrated low incidence of side effects such as rebound redness and loss of efficacy over time with Lumify Preservative Free eye drops. The company plans its availability in early 2025 as single-use vials 

• Lumify is a free preservative eye drop that contains OTC brimonidine tartrate’s low dose 0.025% for treating ocular redness 

5. X4 Pharmaceuticals’ Xolremdi Receives the US FDA’s Approval for the Treatment of WHIM Syndrome 

               Active Ingredient: Mavorixafor 

               Approved: Apr 26, 2024  

               Company: X4 Pharmaceuticals                      

               Disease: WHIM syndrome 

• The US FDA has approved Xolremdi (mavorixafor) capsules for treating WHIM syndrome patients (12yrs. & above) and granted a Rare Pediatric Disease Priority Review Voucher to the company 

• The approval was supported by the P-III (4WHIM) study assessing the safety & efficacy of Xolremdi vs PBO to treat WHIM syndrome patients (n=31, 12yrs. & above) 

• The study depicted an increase in time above threshold for absolute neutrophil count (TAT-ANC) & absolute lymphocyte count (TAT-ALC), total infection score reduction by ~40% and an annualized infection rate reduction by 60% with no difference in total wart change scores over 52wks. 

6. Pfizer’s BEQVEZ (fidanacogene elaparvovec-dzkt) Receives US FDA Approval for Adults with Hemophilia B 

               Active Ingredient: Fidanacogene elaparvovec-dzkt 

               Approved: Apr 26, 2024  

               Company: Pfizer 

               Disease: Haemophilia B 

• US FDA granted approval to Pfizer’s Beqvez, based on BENEGENE-2 (P-III) study (n=45) to treat of Haemophilia B adult patient using factor IX (FIX) prophylaxis therapy, with life-threatening hemorrhage, spontaneous bleeding episodes, no FDA-approved test detected neutralizing antibodies to AAVRh74var capsid; met 1EP of non-inferiority in the ABR of total bleeds 

• Based on durability response of patients to Beqvez, Pfizer launching a warranty program to provide greater certainty to payers, maximize access for eligible patients, and financial protection against risk of efficacy failure 

• BEQVEZ, a one-time treatment enables haemophilia B patients to produce FIX themselves rather than FIX SOC (IV; multiple times wk or mos); awaits assessment by EMA; gained regulatory clearance in Canada 

7. Aquestive Therapeutics’ Libervan Buccal Film Receives the US FDA’s Approval to Treat Seizure Activity  

               Active Ingredient: Diazepam 

               Approved: Apr 26, 2024  

               Company: Aquestive Therapeutics 

               Disease: Seizure Activity (Seizure Clusters, Acute Repetitive Seizures) 

• Aquestive’s Libervant buccal film (oral, 5mg, 7.5mg, 10mg, 12.5mg & 15mg) received the US FDA’s approval as an acute treatment of intermittent, stereotypic episodes of frequent seizure activity (seizure clusters, acute repetitive seizures), distinct from the usual pattern in epilepsy patients (2-5yrs.) 

• The company further provided an update on Anaphylm (epinephrine) sublingual film, mentioning, its development is under progress for NDA submission, expected during the end of 2024    

• The company is accepting & filling non-Medicaid prescriptions for libervant and will expand distribution in the upcoming wks. Libervant is a film formulation of diazepam that is administered buccally and developed as an acute treatment of frequent seizure episodes 

Note: According to the FDA's April 2024 approval list, following drugs were also approved; however, no PR was available: 

• Xromi (Apr 04, 2024) 
• Lumisight (Apr 17, 2024) 
• Rezenopy (Apr 19, 2024) 
• Pivya (Apr 24, 2024) 
• Vasopressin (Apr 30, 2024)    

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