Shots:
- The EMA’s CHMP has granted positive opinions and approvals to 4 Biologics and 4 New Chemical Entites in April 2025, leading to treatments for patients and advances in the healthcare industry
- The major highlighted drugs were Amgen’s Tepezza to Treat Thyroid Eye Disease
- PharmaShots has compiled a list of 8 drugs that have been granted positive opinions and approvals by the EC, respectively
Company: Eisai, Biogen and BioArctic
Product: Leqembi
Active Ingredient: Lecanemab
Disease: Alzheimer’s Disease
Date: Apr 15, 2025
Shots:
- The EC has approved Leqembi for the treatment of early Alzheimer’s disease in all 30 EEA states
- Eisai handles global development & regulatory filings for Leqembi, co-commercializing & co-promoting it with Biogen while retaining final decision-making authority. Eisai co-promotes Leqembi with Biogen in the EU (excl. Nordics) & with BioArctic in the Nordic countries, distributing it as MA Holder in all regions
- Leqembi is a humanized anti-soluble aggregated Aβ mAb that selectively binds to Aβ plaque components, reducing Aβ protofibrils & plaques in the brain
Company: Regeneron
Product: Lynozyfic
Active Ingredient: Linvoseltamab
Disease: R/R Multiple Myeloma
Date: Apr 23, 2025
Shots:
- The EC has granted conditional approval to Lynozyfic for the treatment of adults with r/r multiple myeloma based on P-I/II (LINKER-MM1) trial data assessing Lynozyfic in >300 pts; P-II dose expansion portion is ongoing. BLA under the US FDA’s Review (PDUFA: Jul 10, 2025)
- In the trial, 117 pts on 200mg had a 71% ORR, with 50% CR rate per IRC, where CR or stringent CR pts showed MRD negativity rate of 41%; trial showed mDOR of 29mos.
- Also, Linvoseltamab is being studied in P-III (LINKER-MM3) trial as a monotx. & P-Ib (LINKER-MM2) trial to treat r/r MM in combination with other cancer treatments
Company: Jazz Pharmaceuticals
Product: Ziihera
Active Ingredient: Zanidatamab
Disease: Biliary Tract Cancer
Date: Apr 25, 2025
Shots:
- The CHMP has recommended conditional marketing approval of zanidatamab for the treatment of inoperable locally advanced or metastatic HER2+ (IHC 3+) biliary tract cancer (BTC) adults, who were previously treated with ≥1L of therapy; EC’s potential approval will be valid in 30 EEA states
- Opinion was based on P-IIb (HERIZON-BTC-01) trial to assess anti-tumor activity of zanidatamab alone in HER2+ inoperable & advanced or metastatic BTC
- Zanidatamab received the US FDA’s accelerated approval in Nov 2024 for previously treated BTC based on ORR & duration of response; continued approval depends on confirmation of clinical benefit in the ongoing P-III (HERIZON-BTC-302) trial
Company: Vertex Pharmaceuticals
Product: Alyftrek
Active Ingredient: Deutivacaftor/Tezacaftor/Vanzacaftor
Disease: Cystic Fibrosis
Date: Apr 25, 2025
Shots:
- The CHMP has recommended Alyftrek (deutivacaftor/tezacaftor/vanzacaftor) to treat patients (≥6yrs.) with cystic fibrosis (CF), having at least one non-class I mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. Alyftrek is approved in the US and UK and is under review in Canada, Switzerland, Australia, and New Zealand
- Vanzacaftor & tezacaftor help increase CFTR protein at the cell surface, while deutivacaftor enhances its function by boosting channel open probability to improve salt and water flow
- In trials, Alyftrek matched Kaftrio (ivacaftor/tezacaftor/elexacaftor) on ppFEV1 and outperformed it in reducing sweat chloride
Company: Italfarmaco
Product: Duvyzat
Active Ingredient: Givinostat
Disease: Duchenne Muscular Dystrophy
Date: Apr 25, 2025
Shots:
- Opinion for Duvyzat to treat ambulant DMD pts (≥6yrs.) with corticosteroids is based on P-III (EPIDYS) study assessing Duvyzat (BID) vs PBO in ambulant boys (n=179); EC’s decision expected in Jul 2025
- Trial met its 1EP by improving four-stair climb completion time, with benefits seen in 2EPs (NSAA & MRI fat infiltration) & a 40% decrease in cumulative NSAA item loss; data was published in The Lancet Neurology. Ongoing extension trial data showed delay in DMD progression, with median ambulation loss at 18.1 vs 15.2yrs. for controls; data presented at MDA conference
- Duvyzat is being evaluated in 2 trials: P-III to determine its safety & efficacy in non-ambulant DMD pts (≥9yrs.) & P-II to assess safety + early treatment benefits in DMD pts (2-5yrs.)
Company: PTC Therapeutics
Product: Sephience
Active Ingredient: Sepiapterin
Disease: Phenylketonuria
Date: Apr 25, 2025
Shots:
- The CHMP has issued a positive opinion recommending marketing authorization for Sephience (sepiapterin) to treat PKU in patients of all ages and disease severities, expected the EC approval within two mos., with the decision applying across the EU including Iceland, Norway, and Liechtenstein
- Sepiapterin’s NDA is under FDA review (PDUFA: July 29, 2025), and approval reviews are underway in countries like Japan and Brazil
- Sephience is an oral synthetic sepiapterin with a dual mechanism that boosts phenylalanine hydroxylase (PAH) enzyme activity
Company: Purpose Pharma
Product: Attrogy
Active Ingredient: Diflunisal
Disease: Hereditary Transthyretin-mediated Amyloidosis
Date: Apr 25, 2025
Shots:
- The CHMP has recommended Attrogy (diflunisal) to treat hereditary transthyretin-mediated amyloidosis (hATTR amyloidosis) in adult pts. with stage 1 or stage 2 polyneuropathy
- The EC will make decision within 67 days, and if approved, Attrogy will be authorized in all EU & EEA countries Iceland, Lichtenstein and Norway
- Diflunisal has received EU ODD for ATTR amyloidosis, with a US application submitted for ODD
Company: Amgen
Product: Tepezza
Active Ingredient: Teprotumumab
Disease: Thyroid Eye Disease
Date: Apr 25, 2025
Shots:
- The EMA has recommended marketing authorization for Tepezza (teprotumumab) for adults with mod. to sev. thyroid eye disease
- The opinion was based on three randomized, PBO-controlled studies in 225 active and 62 chronic TED pts. After 24 wks., Tepezza significantly reduced eye protrusion (−2 to −2.3 mm) in protrusion of the eyeball from the eye socket and CAS in active TED, with a smaller effect (−1.5 mm) in chronic cases
- Tepezza (mAb) blocks autoimmune activation of orbital fibroblasts to help prevent and slow TED progression
Related Post: Insights+: EMA Marketing Authorization of New Drugs in March 2025
