Shots:
- The EMA approved 8 New Chemical Entity (NCE) and 3 Biologic Drugs in April 2023, leading to treatments for patients and advances in the healthcare industry
- In April 2023, the major highlights drugs were Rinvoq’s Approval for active Crohn’s Disease, Opzelura for non-segmental vitiligo with facial involvement in adults and adolescents
- PharmaShots has compiled a list of a total of 11 new drugs approved by the EMA in April 2023
Breyanzi
Active ingredient: lisocabtagene maraleucel Approved: April 01, 2023
Company: BMS Disease: Large B-cell Lymphoma
- The EMA’s CHMP has recommended the approval of Breyanzi for adult patients with DLBCL, HGBCL, PMBCL & FL3B who relapsed within 12mos. from completion of, or are refractory to 1L chemoimmunotherapy
- The opinion was based on the P-III study (TRANSFORM) evaluating Breyanzi vs SoC consisting of salvage CT, followed by high-dose CT + HSCT in 184 patients. The EC’s final decision is expected within ~2mos. following receipt of the CHMP opinion
- The EC’s decision will be valid to all EU member states, Iceland, Norway, and Liechtenstein. Breyanzi is a CD19-directed CAR T cell therapy with a 4-1BB costimulatory domain & was approved in Japan for 2L treatment of r/r LBCL & in Japan, EU, Switzerland & Canada for r/r LBCL
Ultomiris
Active ingredient: ravulizumab Approved: April 03, 2023
Company: AstraZeneca Disease: Neuromyelitis Optica Spectrum Disorder
- The EMA’s CHMP has recommended Ultomiris for marketing authorization in the EU for adult patients with NMOSD who are anti-aquaporin-4 Ab+
- The opinion was based on the P-III trial (CHAMPION-NMOSD) evaluating Ultomiris in 58 patients across North America, the EU, Asia-Pacific & Japan. The trial met its 1EPs of time to first on-trial relapse as confirmed by an independent adjudication committee & showed zero relapses with a median treatment duration of 73wks. (relapse risk reduction 98.6%) & continuing through a median duration of 90wks.
- The safety & tolerability were consistent with prior studies and real-world use with no new safety signals. The regulatory submissions for Ultomiris are under review with multiple health authorities, incl. in the US & Japan
Rinvoq
Active ingredient: upadacitinib Approved: April 17, 2023
Company: AbbVie Disease: Active Crohn’s Disease
- The approval was based on 3 P-III trials incl. (U-EXCEED & U-EXCEL) induction studies & (U-ENDURE) maintenance study to evaluate upadacitinib (45mg, qd) as IT & (15/30mg, qd) as MT vs PBO in adults
- The results showed that more patients achieved the co-1EPs in the induction & maintenance study treated with Rinvoq 45mg @12wk. and (15 & 30mg) @52wks. demonstrated an endoscopic response (35% & 46% vs 4% & 13%) and (28% & 40% vs 7%); clinical remission (40% & 51% vs 14% & 22%) and (36% & 46% vs 14%), respectively
- In the 2EPs & additional EPs from the IT & MT, corticosteroid-free clinical remission (37% & 44% vs 7% & 13%) and (35% & 45% vs 14%); mucosal healing (17% & 25% vs 0% & 5%) and (13% & 24% vs 4%). The safety profile was consistent with the known safety profile of Rinvoq
Opzelura
Active ingredient: ruxolitinib Approved: April 21, 2023
Company: Incyte Disease: Non-Segmental Vitiligo
- The EC has granted marketing authorization for Opzelura (15mg/g) to treat adults & adolescents aged ≥12yrs. The EC decision was based on the P-III trials (TRuE-V1 & V2) evaluating Opzelura vs vehicle in 600+ patients aged ≥12yrs.
- The results showed an improvement in facial & total body repigmentation as shown by the no. of patients who reached F-VASI-T-VASI EPs @24wk. & in an OLE @52wk.
- ≥75% improvement from baseline in F-VASI75 in both studies (29.8% & 30.9% vs 7.4% & 11.4%), respectively while one in two patients achieved F-VASI75 @52wk. & one in three @52wk., ≥15% vs ~2% achieved ≥90% improvement in F-VASI (F-VASI90) @24wk., no serious TRAEs related to ruxolitinib were reported. The EC’s decision applies to all 27 EU Member States, Iceland, Norway & Liechtenstein
Akeega
Active ingredient: niraparib & abiraterone acetate Approved: April 21, 2023
Company: Janssen Disease: Metastatic Castration-Resistant Prostate Cancer
- The EC has granted marketing authorization for Akeega in the form of a dual-action tablet for mCRPC. The authorization was based on the P-III trial (MAGNITUDE) evaluating niraparib + AA & prednisone or prednisolone vs PBO + abiraterone acetate & prednisone in 765 patients
- The results showed an improvement in rPFS in all HRR+ patients with 47% risk reduction in patients with BRCA1/2 gene mutations. At the median follow-up at 24.8mos. in the BRCA subgroup, rPFS showed a consistent & clinical treatment effect with m-rPFS (19.5 vs 10.9mos.)
- A trend towards improved OS, improvement in TSP & clinical improvement in time-to-initiation of cytotoxic CT. The safety profile was consistent with the known safety profile of each agent, grade 3/4 AEs in patients with HRR gene alterations (67% vs 46.4%) with maintained overall QoL
Vafseo
Active ingredient: vadadustat Approved: April 26, 2023
Company: Akebia Disease: Symptomatic Anaemia
- The approval was based on the results from a comprehensive development program of ~7,500 patients, incl. the P-III (INNO2VATE) program of vadadustat (HIF-PH inhibitor) for the treatment of anemia due to CKD in adult patients on dialysis
- In each of the two (INNO2VATE) studies, vadadustat met the primary & secondary efficacy EPs which was found to be non-inferior to darbepoetin alfa as measured by a mean change in Hb b/w baseline & primary evaluation period (24-36wk.) and secondary evaluation period (40-52wk.)
- The therapy also achieved the primary safety EPs & was non-inferior to darbepoetin alfa in time to 1st occurrence of major adverse cardiovascular EPs. The approval is valid for all 27 EU member states, Iceland, Norway & Liechtenstein
Cosentyx
Active ingredient: secukinumab Approved: April 26, 2023
Company: Novartis Disease: Hidradenitis Suppurativa
- The EMA’s CHMP has adopted a positive opinion recommending marketing authorization of Cosentyx. The opinion was based on the P-III trials (SUNSHINE) & (SUNRISE) evaluating Cosentyx (300mg, q2w/q4w) vs PBO in 545 & 544 patients across 40 countries
- In both trials, patients treated with Cosentyx (300mg, q2w) achieved HiSCR (45.0% vs 33.7%) & (42.3% vs 31.2%) @16wks.; (46.1% vs 31.2%) & (41.8% vs 33.7%) with Cosentyx (300mg, q4w). Treatment response rates continued to improve beyond the 1EPs analysis @16wk.
- ≥55% achieved a HiSCR measure @52wk., ≥50% reduction in pain. The safety results were consistent with the well-established profile of Cosentyx & have been submitted to the US FDA with decisions expected in 2023
Opfolda
Active ingredient: miglustat Approved: April 26, 2023
Company: Amicus Therapeutics Disease: Late-Onset Pompe Disease
- The EMA’s CHMP has adopted a positive opinion recommending marketing authorization of miglustat. The EC’s decision & commercial launch of Pombiliti + Opfolda is expected in Q3’23
- The opinion was based on the P-III trial (PROPEL) evaluating the efficacy, safety & tolerability of Pombiliti. AT-GAA showed improvements in musculoskeletal & respiratory measures in clinical studies
- If Pombiliti + Opfolda is approved, it will be the first two-component therapy available in the EU for the treatment of adults with LOPD. Pombiliti + Opfolda also known as AT-GAA, the two-component investigational therapy that combines cipaglucosidase alfa to enable high-affinity uptake through the M6P receptor
Columvi
Active ingredient: glofitamab Approved: April 26, 2023
Company: Roche Disease: Diffuse Large B-Cell Lymphoma
- The EMA’s CHMP has recommended the approval of Columvi for adult patients with r/r DLBCL after 2 lines of systemic therapy. The EC’s final decision is expected in the near future
- The recommendation was based on the pivotal cohort in the P-I/II (NP30179) study evaluating Columvi. The results showed that Columvi given as a fixed course induced a CR (35.2%), ORR (50%) while 74.6% continued to experience a response @12mos. who achieved CR, the median duration of CR (not reached), and the median follow-up for DoR was 12.8mos. & median time to first CR was 42 days
- 1 patient discontinued treatment due to CRS while the NEJM provided additional data from a larger cohort in the (NP30179) study. Columvi was approved in Health Canada for r/r DLBCL
Camzyos
Active ingredient: mavacamten Approved: April 27, 2023
Company: BMS Disease: Obstructive Hypertrophic Cardiomyopathy
- The EMA’s CHMP has recommended approval of Camzyos (cardiac myosin inhibitor) for symptomatic (New York Heart Association, NYHA, class II-III) obstructive HCM
- The opinion was based on 2 P-III trials (EXPLORER-HCM) & (VALOR-HCM) evaluating Camzyos vs PBO in 251 & 112 adult patients. Both trials met their 1EPs & 2EPs & showed a clear treatment effect in the P-III trial (EXPLORER-HCM) with clinical improvements in exercise capacity, symptoms, and functional status along with an improvement in LVOTO
- The P-III trial (VALOR-HCM) showed an improvement across cardiac measures who met the 2011 ACC/AHA or 2014 ESC guideline criteria for SRT & were referred for an invasive procedure. The therapy also showed a reduction in need or eligibility for SRT
Orkambi
Active ingredient: umacaftor/ivacaftor Approved: April 27, 2023
Company: Vertex Disease: Cystic Fibrosis
- The EMA’s CHMP has adopted a positive opinion for the label extension of Orkambi (lumacaftor/ivacaftor) to treat children with CF aged 1-<2yrs. who have two copies of the F508del mutation in the CFTR gene
- The expanded approval was based on the P-III clinical trial evaluating Orkambi in children aged 1-<2yrs. which was found to be safe and well tolerated, and reduced the sweat chloride concentration
- Orkambi was approved in the EU for the treatment of patients with CF aged ≥2yrs. who have two copies of the F508del mutations. If Orkambi is approved, a treatment option will be available for the first time for about 300 young children with CF
Note: Breyanzi, Ultomiris, Cosentyx, Opfolda & Orkambi received EMA’s CHMP Positive Opinion & EC’s Marketing Authorization for Akeega, Vafseo while CHMP Recommendation for Approval of Columvi
Related Post: Insights+: EMA Marketing Authorization of New Drugs in March 2023
