Shots:
- The EMA approved 5 BLA and 8 New Chemical Entities in February 2024, leading to treatments for patients and advances in the healthcare industry
- In February 2024, the major highlighted drugs were Reblozyl to treat Transfusion-Dependent Anemia and Velsipity for the treatment of Severely Active Ulcerative Colitis
- PharmaShots has compiled a list of a total of 8 new drugs approved by the EMA in February 2024
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Product Name: Velsipity
Active ingredient: Etrasimod
Company: Pfizer
Date: Feb 20, 2024
Disease: Ulcerative Colitis
Shots:
- The positive opinion was granted by CHMP based on the results from the P-III (CARTITUDE-4) clinical trial evaluating the safety & efficacy of Carvykti vs Standard Therapy (Pomalidomide, Bortezomib & Dexamethasone (PVd)/Daratumumab, Pomalidomide and Dexamethasone (DPd)) in patients with r/lenalidomide-r multiple myeloma
- Patients in the trial have previously received 1-3 prior lines of therapy, depicted disease progression on the last therapy & were refractory to lenalidomide
- Carvykti is a BCMA-directed genetically modified autologous T-cell immunotherapy that has received a CMA from the EMA in May 2022 & approval from the US FDA in Feb 2022 for the treatment of r/r MM
Product Name: Qalsody
Active ingredient: Tofersen
Company: Biogen
Date: Feb 23, 2024
Disease: Amyotrophic Lateral Sclerosis (ALS)
Shots:
- The opinion was based on the results from the P-III (VALOR) trial evaluating the safety & efficacy of Qalsody (100mg) vs PBO in ALS patients (n=108) associated with SOD1 mutations. The 1EPs of the study include changes in ALSFRS-R total score from baseline to wk.28 & 2EPs includes change in total cerebrospinal fluid SOD1 protein concentration, plasma NfL, slow vital capacity & handheld dynamometry in 16 muscles
- The results depicted a 60% reduction in plasma NfL & an improvement in physical abilities measured by ALSFRS-R total score with Qalsody vs PBO
- Qalsody, an antisense oligonucleotide (ASO), binds to SOD1 mRNA to reduce SOD1 protein production. Additionally, Qalsody received the US FDA’s accelerated approval for ALS
Product Name: Carvykti
Active ingredient: Ciltacabtagene autoleucel; Cilta-cel
Company: Janssen Pharmaceuticals
Date: Feb 23, 2024
Disease: Multiple Myeloma (MM)
Shots:
- The positive opinion was granted by CHMP based on the results from the P-III (CARTITUDE-4) clinical trial evaluating the safety & efficacy of Carvykti vs Standard Therapy (Pomalidomide, Bortezomib & Dexamethasone (PVd)/Daratumumab, Pomalidomide and Dexamethasone (DPd)) in patients with r/lenalidomide-r multiple myeloma
- Patients in the trial have previously received 1-3 prior lines of therapy, depicted disease progression on the last therapy & were refractory to lenalidomide
- Carvykti is a BCMA-directed genetically modified autologous T-cell immunotherapy that has received a CMA from the EMA in May 2022 & approval from the US FDA in Feb 2022 for the treatment of r/r MM
Product Name: Keytruda
Active ingredient: Pembrolizumab
Company: Merck
Date: Feb 23, 2024
Disease: Non-Small Cell Lung Cancer (NSCLC)
Shots:
- The company received the positive opinion based on the results from the P-III (KEYNOTE-671) clinical trial evaluating neoadjuvant Keytruda + platinum-containing CT followed by adjuvant Keytruda monotx. vs neoadjuvant PBO in patients with resectable NSCLC at high risk of recurrence
- The study met its dual 1EP by demonstrating a statistically significant & clinically meaningful improvement in OS and EFS. Additionally, based on these results the company expects to receive EC’s decision by H1’24
- Earlier in Oct 2023, Keytruda in combination with platinum-containing CT received the US FDA’s approval for the treatment of patients with resectable (tumors ≥4 cm or node-positive) NSCLC
4. Vertex Receives CHMP’s Positive Opinion for Kalydeco as a Treatment of Cystic Fibrosis in Infants
Product Name: Kalydeco
Active ingredient: Ivacaftor
Company: Vertex Pharmaceuticals
Date: Feb 23, 2024
Disease: Cystic Fibrosis
Shots:
- The company received the CHMP’s positive opinion for the label expansion of Kalydeco (Ivacaftor) to include the treatment of infants (aged 1mos.-4mos.) with cystic fibrosis with specific mutations in the CFTR gene (R117H/G551D/G1244E/G1349D/G178R/G551S/S1251N/S1255P/S549N/S549R)
- Kalydeco has been approved by the EU for the treatment of cystic fibrosis patients aged ≥4mos. with specific CFTR gene mutations
- Kalydeco is an oral medicine designed to enhance the ability of the CFTR protein to transport salt and water across the cell membrane thereby hydrating and clearing mucus from the airways
Product Name: Reblozyl
Active ingredient: Luspatercept
Company: Bristol Myers Squibb
Date: Feb 26, 2024
Disease: Transfusion-Dependent Anemia Due to Myelodysplastic Syndrome (MDS)
Shots:
- The positive opinion was supported by the P-III (COMMANDS) study assessing the safety & efficacy of Reblozyl (1.0mg/kg, titration ~1.75mg/kg, Q3W) vs epoetin alfa (450 IU/kg, titration ~1050IU/kg, QW) for ≥24 wks. to treat transfusion-dependent anemia due to very low-, low- or intermediate-risk (IPSS-R) MDS in patients naïve to erythropoiesis-stimulating agent
- The data, as of Mar 2023, in patients (n=363) depicted RBC-TI of at least 12wks. in 60.4% vs 34.8% with mean Hb increase of ~1.5 g/dL in first 24wks., 74.2% vs 53% had HI-E increase of at least 8wks., 68.1% vs 48.6% experienced RBC-TI of at least 12wks.& a DoR of 128.1wks. vs 89.7wks.
- The safety profile was consistent with the prior MDS programs
Product Name: Voydeya
Active ingredient: Danicopan
Company: AstraZeneca
Date: Feb 26, 2024
Disease: Paroxysmal Nocturnal Haemoglobinuria (PNH) with Residual Haemolytic Anaemia
Shots:
- The CHMP has recommended Voydeya as an add-on therapy to SoC for treating PNH adult patients with residual haemolytic anaemia
- The recommendation was based on the P-III (ALPHA) study investigating the safety & efficacy of Voydeya as an add-on therapy to Ultomiris or Soliris (SoC) for treating PNH patients with extravascular haemolysis (EVH)
- The study achieved its 1EP of change in hemoglobin from baseline to wk. 12 along with the 2EPs incl. transfusion avoidance and change in Functional Assessment of Chronic Illness Therapy – Fatigue (FACIT-Fatigue) score. The drug was well tolerated without any safety concerns. Data from the 12-wk. primary analysis was published in The Lancet Haematology
Product Name: Filspari
Active ingredient: Sparsentan
Company: CSL Vifor and Travere Therapeutics
Date: Feb 26, 2024
Disease: IgA Nephropathy
Shots:
- The CHMP has granted positive opinion to CSL (exclusive commercialization rights holder in the EU, Australia & New Zealand) & Travere’s sparsentan for its conditional approval to treat primary IgAN with a urine protein excretion >1.0 g/day. EC’s decision is anticipated in Q2’24
- The opinion was supported by the P-III (PROTECT) trial investigating the safety & efficacy of sparsentan (400mg) vs irbesartan (300mg) in IgAN patients (n=404, 18yrs. of age & above) with persistent proteinuria after receiving MTD and at least -50% of maximum labelled dose of ACE/ARB therapy
- The results depicted fast & sustained proteinuria reduction, ability of sparsentan to preserve kidney function & delay time to kidney failure
Product Name: Tevimbra
Active ingredient: Tislelizumab
Company: BeiGene
Date: Feb 27, 2024
Disease: Non-Small Cell Lung Cancer (NSCLC)
Shots:
- The MAA was based on the evaluation of NSCLC patients (n=1,499) in 3 P-III studies incl. (RATIONALE 307) for Tevimbra + carboplatin + paclitaxel/nab-paclitaxel vs CT (1L treatment for sq. NSCLC), (RATIONALE 304) for Tevimbra + pemetrexed + platinum-containing CT vs CT (1L treatment for non-sq. NSCLC) & (RATIONALE 303) for Tevimbra vs CT (locally advanced/metastatic NSCLC)
- At 8.6mos. follow-up, (RATIONALE 307) showed a PFS of 7.6 & 7.6 vs 5.5mos. & ORR of 72.5 & 74.8 vs 49.6%, at 9.8mos. follow-up (RATIONALE 304) showed a PFS of 9.7 vs 7.6mos & (RATIONALE 303) showed an OS of 17.2 vs 11.9mos.
- Tevimbra, an IgG4 anti-PD-1 mAb, received the EC’s approval for ESCC in 2023 & is under review by the US FDA for ESCC
Note:
According to the EMA’s February 2024 approval list, Seqirus Netherlands B.V.’s Incellipan and Celldemic, Incyte Biosciences’ Zynyz, Pfizer’s Cibinqo, Nova Laboratories’ Xromi was also approved; however, no PR was available
Related Post: Insights+: EMA Marketing Authorization of New Drugs in January 2024
