Insights+: EMA Marketing Authorization of New Drugs in March 2024

Shots:

The EC approved 1 Biologic and 11 New Chemical Entities in March 2024, leading to treatments for patients and advances in the healthcare industry
In March 2024, the major highlighted drugs were Novartis’ Fabhalta to treat PNH and UCB’s Bimzelx for the treatment of Adults with Moderate to Severe Hidradenitis Suppurativa
PharmaShots has compiled a list of 6 drugs that were approved or have been granted positive opinion by the EC or EMA’s CHMP, respectively

1. Novo Nordisk’s Awiqli Receives the CHMP’s Positive Opinion for the Treatment of Diabetes

Product Name: Awiqli

Active ingredient: Insulin icodec

Company: Novo Nordisk

Date: Mar 21, 2024

Disease: Diabetes

Shots:

Novo Nordisk’s Awiqli (QW basal insulin icodec) has received CHMP’s positive opinion and recommending it for the final approval which is anticipated in next ~67days/ ~2mos
The opinion was supported by the six P-IIIa (ONWARDS) study assessing the safety & efficacy of Awiqli in type 1 & type 2 diabetes adults (n= >4,000) incl. Real-world analysis
The study showed superior blood sugar reduction (by HbA1c) & Time in Range (time spent in RBS range) among T2D patients. Awiqli reduced HbA1c, but had a higher hypoglycemia rate

2. Outlook Therapeutics’ Lytenava Gains the CHMP’s Positive Opinion for the Treatment of Wet AMD

Product Name: Lytenava

Active ingredient: Bevacizumab-vikg

Company: Outlook Therapeutics

Date: Mar 22, 2024

Disease: Wet Age-related Macular Degeneration

Shots:

Outlook’s Lytenava has received CHMP’s positive opinion to treat Wet AMD. If approved Lytenava expected to gain 10 yrs of market exclusivity in EU
The opinion was supported by 3 wet AMD clinical programs (NORSE ONE, NORSE TWO & NORSE THREE) assessing ONS-5010 along with retrospective bibliographic literature
Lytenava, an ophthalmic formulation of bevacizumab, is being developed as an intravitreal injection for treating wet AMD and other retinal diseases. It is an ophthalmic formulation of existing bevacizumab which has been off label used in wAMD globally

3. Novartis Reports the CHMP’s Positive Opinion for Fabhalta (iptacopan) to Treat Paroxysmal Nocturnal Hemoglobinuria (PNH)

Product Name: Fabhalta

Active ingredient: Iptacopan

Company: Novartis

Date: Mar 22, 2024

Disease: Paroxysmal Nocturnal Hemoglobinuria

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The opinion was based on the P-III (APPLY-PNH) & (APPOINT-PNH) trials of Fabhalta (200mg, oral, BID) in PNH patients with residual anemia who switched from Anti-C5 and complement inhibitor naïve (incl. anti-C5 therapies) respectively for 24wks.
In the APPLY-PNH study, 82.3% vs 2.0% had a ≥2 g/dL increase in Hb levels without transfusions while in APPOINT-PNH, 92.2% of complement inhibitor-naïve Fabhalta patients reached the same
The transfusion avoidance rate of 94.8% vs 25.9% was observed in APPLY-PNH study. Fabhalta controlled IVH with mean LDH levels <1.5 x upper limit of normal and patients experienced fatigue improvements based on FACIT-F scores

4. UCB’s Bimzelx Receives CHMP’s Positive Opinion to Treat Moderate to Severe Hidradenitis Suppurativa

Product Name: Bimzelx

Active ingredient: Bimekizumab

Company: UCB

Date: Mar 22, 2024

Disease: Moderate to Severe Hidradenitis Suppurativa

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The recommendation was supported by the P-III studies (BE HEARD I and BE HEARD II) assessing the safety and efficacy of Bimzelx 1014 patients with PsA evaluating bimekizumab vs. PBO at week 16
Both trials met their 1EPs & 2EPs measured by HiSCR50 and HiSCR75 respectively at wk. 16. Clinical responses were sustained up to 48wk. in (BE HEARD I and BE HEARD II) trial
For its final decision, the EC will refer to bimekizumab's positive opinion grant by the CHMP on moderate to severe HS. The marketing authorization would apply to all the EU member states and countries of the EEA

5. Norgine’s NPJ5008 (Dantrolene sodium hemiheptahydrate) Receives CHMP’s Positive Opinion for the Treatment of Malignant Hyperthermia

Product Name: NPJ5008

Active ingredient: Dantrolene sodium hemiheptahydrate

Company: Norgine

Date: Mar 22, 2024

Disease: Malignant Hyperthermia

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Norgine received a positive CHMP opinion for NPJ5008 (Dantrolene sodium hemiheptahydrate) for the treatment of Malignant Hyperthermia
With NPJ5008, dantrolene is prepared and administered quickly using novel formulation vs. Dantrolene IV (dantrolene sodium). NPJ5008 (IV) contains 120 mg of dantrolene in each vial and is reconstituted in 20 mL of water
For its final decision, the EC will refer to NPJ5008's positive opinion grant by the CHMP. NPJ5008’s UK (MHRA) & Switzerland (Swiss Medic) submission is ongoing

6. Pfizer’s Emblaveo (aztreonam-avibactam) Receives Positive CHMP Opinion for the Treatment of Patients with Multidrug-Resistant Infections

Product Name: Emblaveo

Active ingredient: Aztreonam-avibactam

Company: Pfizer

Date: Mar 22, 2024

Disease: Adult patients with cIAI, HAP incl. VAP, and cUTI, incl. pyelonephritis.

Shots:

Pfizer’s Emblaveo has received a positive CHMP opinion for the treatment of patients with cIAI, HAP (incl. VAP), and cUTI (incl. Pyelonephritis).
The MAA was supported by results from the P-III study comprising the REVISIT and ASSEMBLE studies evaluating the efficacy, safety, and tolerability of Emblaveo in treating serious bacterial infections due to Gram-negative bacteria (incl. MBL-producing multidrug-resistant pathogens for which there are limited or no treatment options)
AbbVie and Pfizer jointly developing Emblaveo, where Pfizer holds the global rights to commercialize Emblaveo outside of the U.S. and Canada and the development was also funded by US DoHHS.

Note:

According to the EMA’s March 2024 approval list, Neuraxpharm Pharmaceuticals’ Dimethyl fumarate Neuraxpharm, Celltrion Healthcare’s Omlyclo, Les Laboratoires Servier’s Onivyde pegylated liposomal, Accord Healthcare’s Dimethyl fumarate Accord, BrePco Biopharma’s Neoatricon, and Mylan’s Dimethyl fumarate Mylan was also approved; however, no PR was available