Crovalimab's approval worldwide: When is the new PNH treatment coming to you?

So far, 2024 has been a positive year for patients with paroxysmal nocturnal hemoglobinuria (PNH). With iptacopan's EMA approval and crovalimab's first global approvals becoming a fact in the first half of the year, patients worldwide can benefit from more treatment options.

And while any approval is good news, for patients outside the USA, it may still take a while until the newest treatments for PNH become available. 

Here's what you need to know about the expected timelines for crovalimab's MHRA and EMA approvals, as well as its expected availability in other countries. 

What does crovalimab treat?

Crovalimab (also known by its brand name Piasky) is a prescription medicine for the treatment of paroxysmal nocturnal hemoglobinuria (PNH). The medicine is intended for patients aged 13 and up with a minimum body weight of 40 kg ¹.

Can crovalimab treat sickle cell disease?

Crovalimab is not yet approved as a treatment for sickle cell disease. However, it targets the complement pathway, which is active in sickle cell disease patients. For this reason, crovalimab is currently under investigation for its potential to prevent vaso-occlusive episodes in sickle cell disease patients. The primary results of the phase 2 trial are anticipated in July 2024 ².

How does crovalimab work?

Crovalimab is an anti-C5 monoclonal antibody. It's designed to target a specific complement protein called C5. By blocking its activity, crovalimab stops the process of blood cell destruction, which is characteristic for PNH ³. 

How effective is crovalimab in clinical trials?

Crovalimab's FDA approval was based on the results of the Phase 3 COMMODORE 2 trial. In this trial, patients with PNH were treated either with crovalimab or eculizumab. The key reported results were:

• 79.3% of patients treated with crovalimab achieved hemolysis control, compared to 79% of patients treated with eculizumab;
• 65.7% of crovalimab patients and 68.1% of eculizumab patients avoided red blood cell transfusions;
• The breakthrough hemolysis rates were 10.4% (crovalimab) and 14.5% (eculizumab);
• 63.4% of crovalimab patients achieved stabilized hemoglobin, compared to 60.9% of eculizumab patients ⁴.

Based on these results, crovalimab was found to be comparable to eculizumab in terms of clinical benefits and safety ⁵.

Difference between crovalimab, ravulizumab and eculizumab

All three PNH treatments are anti-C5 monoclonal antibodies. And while they work in similar ways, there are also notable differences between them, including:

• Mechanism of action: Typically, antibodies bind to an antigen only once. However, crovalimab is different in this respect. Unlike ravulizumab and eculizumab, it binds to the antigen repeatedly. This allows it to block the activity of the complement pathway over a longer period of time ⁶.
• Administration: Both ravulizumab and eculizumab are administered via an intravenous infusion. Crovalimab, on the other hand, is administered as a subcutaneous injection. It can even be self-administered. This makes it less burdensome for patients with PNH, as it interferes less with their daily lives ⁷. 

Where is crovalimab approved?

As of June 2024, Piasky (crovalimab) is approved by the FDA in the USA ⁴. However, this is not the only (or first) global approval for the medicine. 

Crovalimab approval in China

Piasky (crovalimab) has been approved in China since February 2024 ⁸. And while it's not typical for novel medicines to get their first global approval in China (usually it's the USA that's faster), in this case it's not a surprise, since it was Roche’s Chinese subsidiary (Chugai Pharmaceutical) that developed crovalimab. Notably, in China, the minimum age for patients to receive crovalimab treatment is 12 years old, which differs from the FDA approved age of 13 ⁸.

Crovalimab approval in Japan

Since March 2024, Piasky (crovalimab) has also been approved in Japan. In Japan, there is no age restriction for starting the treatment with crovalimab, as long as the minimum body weight requirement of 40 kg is met ⁹.

Other global approvals

Crovalimab is currently also being considered for approval in other countries, including Taiwan and Canada.

When will crovalimab get EMA approval?

On 27 June 2024, Piasky (crovalimab) received a positive opinion from the CHMP in Europe. The CHMP is recommending EMA approval for the medicine. And while this clears the road for European approval, it can still take up to 2 months for the approval to become official. 

When will crovalimab be available to European patients?

This is a more difficult question. While Piasky seems likely to become EMA-approved by late August 2024, that won't mean it'll be available across the EU immediately afterwards. 

Every member state follows its own process and timeline for launching newly approved medicines. In some countries, like Germany, this process takes 133 days on average. Other countries, like Romania, can stretch the process to 899 days on average ¹⁰.

In other words, some patients in the EU may already have access to crovalimab by the end of 2024. Others could be facing an additional couple of years of waiting time. 

When will crovalimab be available in the UK?

As of July 2024, Piasky is not yet approved by the MHRA. However, post-Brexit, the MHRA reserves the right to grant "near automatic" approval to medicines after they've been green-lit by the EMA or other trusted regulatory bodies ¹¹. Therefore, at least in theory, the UK approval of crovalimab may not be far off. 

A review by NICE for potential inclusion of crovalimab on the NHS is already underway, with an expected decision date in October 2024 ¹².

If NICE adopts a positive recommendation for including the medicine on the NHS in October, then Piasky could be available to patients in the UK around January-February 2025. Of course, all of this assumes a final EMA approval in August 2024 and its near immediate adoption by the MHRA.

How to get Piasky (crovalimab) before it's available in your country

Although approvals are in motion, not all patients can wait for months or years. If that's your case, you'll be happy to learn that you don't have to wait.

You can access Piasky immediately via the Named Patient Import regulation.

Buy crovalimab as a Named Patient

Not all patients and doctors know that, but you are allowed to buy and import locally unavailable medicines for personal use. Especially if these medicines could improve your quality of life or address life-threatening conditions.

If you want to get crovalimab before its MHRA or EMA approval, this might be the quickest option for you and your doctor.

The regulation that makes this possible is known as the Named Patient Import regulation. To qualify for it, you first need to have a prescription from your treating doctor for a medicine that:

• is approved elsewhere;
• is not approved or available where you live;
• has no local alternatives, and
• is for personal use only.

Already have a prescription? Share it with our team at Everyone.org and we will support you with buying Piasky immediately.

Buy Leqembi

References:

1. HIGHLIGHTS OF PRESCRIBING INFORMATION. Accessdata.fda.gov, Accessed 9 July 2024. 
2. TRIAL IN PROGRESS: THE RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED PHASE 2A CROSSWALK-C TRIAL EVALUATING THE EFFICACY OF CROVALIMAB AS ADJUNCT TREATMENT IN THE PREVENTION OF VASO-OCCLUSIVE EPISODES (VOES) IN PATIENTS (PTS) WITH SICKLE CELL DISEASE (SCD). HemaSphere, 26 April 2023. 
3. Crovalimab for paroxysmal nocturnal hemoglobinuria (PNH). PNH News, 20 March 2024.
4. Ryan, Chris. FDA Approves Crovalimab for Paroxysmal Nocturnal Hemoglobinuria. OncLive, Accessed 9 July 2024.
5. Phase 3 randomized COMMODORE 2 trial: Crovalimab versus eculizumab in patients with paroxysmal nocturnal hemoglobinuria naive to complement inhibition. American Journal of Hematology, 17 June 2024.
6. Crovalimab Approved in China as the First Country, for the Treatment of Paroxysmal Nocturnal Hemoglobinuria (PNH). CHUGAI PHARMACEUTICAL, Accessed 9 July 2024.
7. Crovalimab Offers Effective, Less Burdensome Alternative to Eculizumab for PNH Treatment. American Journal of Managed Care, 30 June 2024.
8. Dunleavy, Kevin. China is the first to approve Roche's PNH drug crovalimab. Fierce Pharma, 8 February 2024.
9. Chugai Obtains Regulatory Approval for “Piasky 340mg” for Paroxysmal Nocturnal Hemoglobinuria in Japan. CHUGAI PHARMACEUTICAL, Accessed 9 July 2024.
10. EFPIA Patients W.A.I.T. Indicator 2021 Survey. Efpia, Accessed 9 July 2024.
11. UK to give “near automatic sign off” for treatments approved by “trusted” regulators. The BMJ, 16 March 2023.
12. Project information | Crovalimab for treating paroxysmal nocturnal haemoglobinuria [ID6140] | Guidance. NICE, Accessed 9 July 2024.