Shots:  

• The EMA granted Positive Opinion to 4 Biologics and 3 New Chemical Entities in June 2024, leading to treatments for patients and advances in the healthcare industry 

• The major highlighted drugs were Merck’s Winrevair to treat Pulmonary Arterial Hypertension (PAH) and Roche’s PiaSky for Paroxysmal Nocturnal Haemoglobinuria (PNH) 

• PharmaShots has compiled a list of 5 drugs that have been granted positive opinion by the EMA’s CHMP 

1. Roche’s PiaSky Receives the CHMP’s Positive Opinion for Treating Paroxysmal Nocturnal Haemoglobinuria (PNH) 

Product Name: PiaSky 

Active ingredient: Crovalimab 

Company: Roche 

Date: June 27, 2024 

Disease: Paroxysmal Nocturnal Haemoglobinuria  

Shots:  

• The CHMP’s positive opinion of PiaSky for PNH adults & adolescents (≥12yrs., 40kg) was based on 3 P-III trials incl. COMMODORE 2 study of PiaSky (SC, Q4W) vs eculizumab (IV, Q2W) in patients not treated with C5 inhibitor; COMMODORE 1 study in subjects switched from C5 inhibitor & COMMODORE 3 study in Chinese subjects new to C5 inhibitor 

• COMMODORE 2 study showed that the drug was superior, well-tolerated & attained disease control with 78% vs 80% AEs 

• PiaSky (C5 protein inhibiting mAb) is currently being studied under 5 P-III clinical evaluations & 3 earlier phase studies to treat complement-mediated diseases such as PNH, atypical haemolytic uremic syndrome & sickle cell disease 

2. Moderna’s mResvia (mRNA-1345) Gains the CHMP’s Positive Opinion to Prevent Lower Respiratory Tract Disease (LTRD) 

Product Name: mResvia 

Active ingredient: mRNA-1345 

Company: Moderna 

Date: June 27, 2024 

Disease: Lower Respiratory Tract Disease 

Shots: 

• The CHMP’s positive opinion of mRESVIA to prevent lower respiratory tract disease (LTRD) due to RSV infection based on the P-III (ConquerRSV) study in adults (n=37,000; ≥60yrs.) 

• Primary analysis (3.7mos. median follow-up) showed 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. Furthermore, the company has filed MAA to other global authorities 

3. Merck’s Winrevair (Sotatercept) Gains the CHMP’s Positive Opinion to Treat Pulmonary Arterial Hypertension (PAH)   

Product Name: Winrevair 

Active ingredient: Sotatercept 

Company: Merck 

Date: June 27, 2024 

Disease: Pulmonary Arterial Hypertension     

Shots: 

• The CHMP has granted positive opinion to Winrevair combined with other therapies to treat PAH. EC’s decision is anticipated in Q3’24, applicable across EU, Iceland, Liechtenstein & Norway 

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

• Winrevair improved 6-minute walk distance (1EP) as well as reduced the death risk from any cause & PAH clinical worsening events (2EPs). Data was published in the NEJM  

4.Regeneron’s Ordspono Gains the CHMP’s Positive Opinion to Treat r/r Follicular Lymphoma (FL) or Diffuse Large B-Cell Lymphoma (DLBCL) 

Product Name: Ordspono 

Active ingredient: Odronextamab 

Company: Regeneron 

Date: June 28, 2024  

Disease: Follicular Lymphoma or Diffuse Large B-Cell Lymphoma   

Shots: 

• The CHMP has granted a positive opinion for conditional marketing authorization of odronextamab (CD20xCD3 bispecific Ab) to treat r/r FL or r/r DLBCL post ≥2L of therapies, with the EC’s decision anticipated in the upcoming mos. 

• The opinion was based on the P-I (ELM-1) & pivotal P-II (ELM-2) studies, showing strong durable response rates & safety in r/r FL or r/r DLBCL adults 

• Regeneron is assessing odronextamab alone & in combinations as earlier lines of therapies for lymphomas, incl. the registrational ELM-1 & ELM-2 trial, P-III (OLYMPIA) study for B-NHLs and early-stage studies with CT-free combinations 

5. Janssen Reports the CHMP’s Positive Opinion of Balversa (Erdafitinib) to Treat Metastatic Urothelial Carcinoma (mUC) 

Product Name: Balversa 

Active ingredient: Erdafitinib 

Company: Janssen 

Date: June 28, 2024  

Disease: Metastatic Urothelial Carcinoma   

Shots: 

• The CHMP has granted a positive opinion to Balversa monotx. (QD, oral) to treat inoperable mUC adults with susceptible FGFR3 mutations & previously treated with at least 1L of therapy with PD-1/PD-L1 inhibitor 

• The opinion was based on the arm 1 of P-III (THOR) trial assessing the safety & efficacy of erdafitinib (n=136) vs CT (n=130) for mUC. Trial was halted, in Jun 2023, at interim analysis with patients randomizing on CT & with an option of crossing over to erdafitinib 

• The study showed an mOS of 12.1mos. vs 7.8mos. at the data cut off, improved mPFS of 5.6mos. vs 2.7mos. and ORR of 35.3% vs 8.5% 

Note: 

According to the EMA’s June 2024 list, the following drugs received the CHMP’s positive opinion; however, no PR was available: 

• Tauvid [flortaucipir (18F)] 

• Eurneffy (epinephrine) 

Related Post: Insights+: EMA Marketing Authorization of New Drugs in May 2024