Shots:   

• The EC has approved to 5 Biologics and 3 New Chemical Entities in August 2024, leading to treatments for patients and advances in the healthcare industry  

• The major highlighted drugs were Johnson & Johnson’s Balversa to treat Metastatic Urothelial Carcinoma (mUC) and Takeda’s Adzynma for Congenital Thrombotic Thrombocytopenic Purpura (cTTP) 

• PharmaShots has compiled a list of 8 drugs that have been approved by the EC 

1. Takeda Reports the EC’s Approval of Adzynma to Treat Congenital Thrombotic Thrombocytopenic Purpura (cTTP) 

Product Name: Adzynma 

Active ingredient: Recombinant ADAMTS13 

Company: Takeda 

Date: Aug 01, 2024  

Disease: Congenital Thrombotic Thrombocytopenic Purpura (cTTP) 

Shots:   

• The EC has approved Adzynma (recombinant ADAMTS13) to treat ADAMTS13 deficiency in cTTP adults & children. Adzynma is also being assessed under P-IIb study for immune-mediated thrombotic thrombocytopenic purpura (iTTP) 

• Approval was based on the P-III study assessing Adzynma to treat cTTP, published in the NEJM, with patients receiving Adzynma (40IU/kg, IV, weekly) or plasma-based therapy for mos.1-6 (period 1), switched treatments for mos.7-12 (period 2) & then Adzynma for mos.13-18 (period 3) 

• Study depicted 0 (Adzynma) vs 1 (plasma-based therapy) acute TTP event and 1 vs 7 (in 6 patients) subacute TTP events in periods 1 & 2. Efficacy data from period 3 was consistent with periods 1 & 2 

2. Moderna’s mRESVIA (mRNA-1345) Vaccine Gains the EC’s Approval to Prevent Lower Respiratory Tract Disease (LTRD) 

Product Name: mRESVIA 

Active ingredient: mRNA-1345 

Company: Moderna 

Date: Aug 22, 2024  

Disease: Lower Respiratory Tract Disease (LTRD)   

Shots:   

• The EC’s approval of mRESVIA (mRNA-1345) vaccine to prevent LTRD due to RSV infection among adults was supported by P-III (ConquerRSV) trial in adults (n=37,000; ≥60yrs.) & is valid across EU plus as Iceland, Liechtenstein & Norway 

• Primary analysis (3.7mos. median follow-up) depicted vaccine efficacy (VE) of 83.7%, published in the NEJM. Supplementary analysis (8.6mos. median follow-up) showed sustained VE of 63.3% against RSV-LRTD incl. ≥2 symptoms with VE of 74.6% & 63% with ≥2 & ≥3 symptoms, respectively 

• In addition, the vaccine received the US FDA’s approval in May 2024 for the same. The company has filed MAA to other global authorities 

3. ARS Pharmaceuticals Reports the EC’s Approval of Eurneffy (Adrenaline Nasal Spray) to Treat Type I Allergic Reactions 

Product Name: Eurneffy 

Active ingredient: Epinephrine 

Company: ARS Pharmaceuticals 

Date: Aug 22, 2024  

Disease: Type I Allergic Reactions (Anaphylaxis) 

Shots:   

• Following the US FDA approval, the EC has approved Eurneffy (2mg) for type I allergic reactions (anaphylaxis) in adults & children (≥30kg). It will be available in Q4’24 through a pharmaceutical company 

• The approval was supported by the results from a study, involving ~1200 administrations among >700 subjects, along with studies and peer-reviewed literature supporting these results 

• The study assessed PK/PD of Eureffy (2mg) under various conditions such as single and repeat dosing, self-administration, pediatric dosing & in nasal conditions like congestion and rhinorrhea due to allergens or infections like cold/flu 

4. Roche’s PiaSky Receives the EC’s Approval to Treat Paroxysmal Nocturnal Haemoglobinuria (PNH) 

Product Name: PiaSky 

Active ingredient: Crovalimab 

Company: Roche 

Date: Aug 22, 2024  

Disease: Paroxysmal Nocturnal Hemoglobinuria (PNH)   

Shots:   

• The EC has granted approval to PiaSky (crovalimab) for treating PNH in adults & adolescents (≥12yrs., weight: ≥40kg) who are either treatment-experienced or treatment-naïve 

• Approval was based on P-III (COMMODORE 2) trial assessing PiaSky vs eculizumab in PNH patients not treated with C5 inhibitors plus results from another P-III trials, COMMODORE 1 (PNH patients switching from C5 inhibitors) & COMMODORE 3 (new to C5 inhibitor treatment in China) 

• COMMODORE 2 showed PiaSky (SC, Q4W) controlled disease, was well-tolerated & non-inferior to eculizumab (C5 inhibitor, given IV, Q2W) with similar safety profiles & AE rates 

5. Johnson & Johnson’s Balversa (Erdafitinib) Receives the EC’s Approval to Treat Urothelial Carcinoma 

Product Name: Balversa 

Active ingredient: Erdafitinib 

Company: Johnson & Johnson 

Date: Aug 22, 2024  

Disease: Metastatic Urothelial Carcinoma (mUC) 

Shots:   

• The EC has approved Balversa (oral, QD) monotx. for treatment-experienced adults with inoperable or metastatic urothelial carcinoma (mUC) having susceptible FGFR3 genetic alterations 

• Approval was supported by results of cohort 1 from the P-III (THOR) trial assessing the safety & effectiveness of Balversa (n=136) vs CT (n=130) to treat mUC with select FGFR alterations and has progressed post previous treatments 

• In Jun 2023, the THOR study was stopped early due to positive interim results, allowing CT patients to switch to Balversa. Erdafitinib showed mOS of 12.1mos. vs 7.8mos. & mPFS of 5.6mos vs 2.7mos., with a confirmed ORR of 35.3% vs 8.5% 

6. Merck’s Winrevair (Sotatercept) Receives the EC’s Approval to Treat Pulmonary Arterial Hypertension (PAH) 

Product Name: Winrevair 

Active ingredient: Sotatercept 

Company: Merck 

Date: Aug 26, 2024  

Disease: Pulmonary Arterial Hypertension (PAH) 

Shots:   

• The EC has granted approval to Winrevair (45 & 60mg) combined with other PAH therapies to treat PAH, valid across whole EU as well as Iceland, Liechtenstein & Norway  

• Approval was based on the P-III (STELLAR) study evaluating the safety & efficacy of Winrevair (target dose 0.7mg/kg, SC, Q3W; n=163) vs PBO (n=160) + stable background therapy in PAH patients (N=323)   

• Winrevair improved the 1EP of 6-minute walk distance of 40.8 meters at 24wks. as well as 2EPs of reducing the death risk from any cause & PAH clinical worsening by 82% (number of events: 7 vs 29) 

7. Regeneron’s Ordspono (Odronextamab) Receives the EC’s Approval to Treat R/R Follicular Lymphoma and Diffuse Large B-cell Lymphoma 

Product Name: Ordspono 

Active ingredient: Odronextamab 

Company: Regeneron 

Date: Aug 26, 2024  

Disease: Follicular Lymphoma & Diffuse Large B-cell Lymphoma (FL & DLBCL) 

Shots:   

• The EC has approved Ordspono for treating r/r FL or r/r DLBCL in patients who have progressed after ≥2L of systemic therapy 

• Approval was supported by Ordspono’s P-I (ELM-1; n=60) trial in patients with CD20+ B-cell malignancies, incl. those who progressed post CAR-T therapy & P-II (ELM-2; n=128) trial for 5 B-cell lymphoma subtypes such as DLBCL, FL, mantle cell lymphoma, marginal zone lymphoma 

• R/R FL patients (ELM-2) had an ORR of 80% & CR in 73% (mDoR: 25mos.); r/r DLBCL patients naïve to CAR T therapy (ELM-2) had an ORR of 52% & CR in 31% (mDoR: 18mos.); r/r DLBCL patients who progressed post CAR-T therapy (ELM-1) had an ORR of 48% & CR in 32% (mDoR: 15mos. 

8. SIFI and Avanzanite Bioscience’s Akantior (Polihexanide) Gains the EC’s Approval to Treat Acanthamoeba Keratitis (AK)     

Product Name: Akantior 

Active ingredient: Polihexanide 

Company: SIFI and Avanzanite Bioscience 

Date: Aug 27, 2024  

Disease: Acanthamoeba Keratitis (AK) 

Shots:   

• Following recommendations from the EMA’s CHMP & COMP, the EC has approved Akantior to treat AK among adults & adolescents (≥12yrs.)  

• Approval was based on the data from P-III (ODAK) study in AK patients (n=135), published in Ophthalmology, demonstrating that the disease cured in 84.8%, full vision restoration was found in 66.7% with none of them requiring optical cornea transplant and 7.5% needed a therapeutic cornea transplant 

• Avanzanite received the exclusive commercialization rights of Akantior in European Economic Area and Switzerland as per an agreement b/w the company and SIFI 

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