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INSIGHTS+

EMA Marketing Authorization of New Drugs in July 2025  

Shots:  The EMA’s CHMP has granted positive opinions and approvals to 3 Biologics and 10 new chemical entities in July 2025, leading to treatments for patients and advances in the healthcare industry   The major highlighted drug was Roche’s Itovebi Secures the EC’s Approval for PIK3CA-mutated Breast Cancer    PharmaShots has compiled a list of 13 drugs…
August 19, 2025

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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 Picture Eisai, Biogen and BioArctic Report the EC’s Approval of Leqembi (lecanemab) for Early Alzheimer’s Disease 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 Regeneron’s Lynozyfic (Linvoseltamab) Receives the EC’s Conditional Approval for R/R Multiple Myeloma 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 Jazz Pharmaceuticals’ Zanidatamab Secures the CHMP Positive Opinion to Treat HER2+ Biliary Tract Cancer (BTC) 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 Vertex Pharmaceuticals’ Alyftrek Receives the CHMP’s Positive Opinion for the Treatment of Cystic Fibrosis 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 Italfarmaco Obtains the CHMP’s Positive Opinion for Conditional Marketing of Duvyzat to Treat Duchenne Muscular Dystrophy (DMD) 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.) PTC Therapeutics’ Sephience Receives the CHMP’s Positive Opinion for the Treatment of Phenylketonuria (PKU) 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 Purpose Pharma’s Attrogy Receives the CHMP’s Positive Opinion for the Treatment of Hereditary Transthyretin-mediated Amyloidosis 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 The CHMP Adopts Positive Opinion on Amgen’s Tepezza to Treat Thyroid Eye Disease 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 February 2025
INSIGHTS+

EMA Marketing Authorization of New Drugs in April 2025  

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…
May 20, 2025

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INSIGHTS+

PharmaShots’ Key Highlights of First Quarter 2025

Shots:   Q1’2025 highlights major acquisitions in the pharma and biotech industry along with clinical trial results and approvals. The talk of the town remained Intra-Cellular Therapies’ acquisition by Johnson & Johnson for ~$14.6B   The quarter highlighted breakthrough results, pivotal clinical data, including Eli Lilly and Incyte's P-III Study Data of Baricitinib to treat Alopecia Areata  PharmaShots brings…
April 1, 2025

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EMA Marketing Authorization of New Drugs in December 2024
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EMA Marketing Authorization of New Drugs in December 2024

Shots:    The EMA’s CHMP has granted positive opinions to 5 Biologics and 4 New Chemical Entities in December 2024, leading to treatments for patients and advances in the healthcare industry   The major highlighted drugs were Galderma’s Nemluvio to treat Prurigo Nodularis and Atopic Dermatitis    PharmaShots has compiled a list of 9 drugs that have been granted positive…
January 23, 2025

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Top Performing Drug – Invega Trinza (March Edition) 
INSIGHTS+

Top Performing Drug – Invega Trinza (March Edition) 

Shots:    In continuation to our previous series on the Top-Performing Drug of the month, based on 2021 revenue, this month we have selected Invega Trinza and prepared an engaging analysis for our readers    Invega Trinza (3-Months injection) is an atypical antipsychotic drug used for the treatment of schizophrenia in patients after adequately being treated with…
March 19, 2024

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