patients traveled an average of 101 kilometers to reach care
Em um país continental onde a distância entre o paciente e o cuidado pode medir centenas de quilômetros, a Anvisa aprovou na segunda-feira o fabhalta, o primeiro medicamento oral para a hemoglobinúria paroxística noturna — uma doença genética rara que transforma o próprio sistema imunológico em inimigo das células sanguíneas. Para os cerca de 1,3 pacientes por milhão afetados anualmente no Brasil, a aprovação representa não apenas uma nova molécula, mas a possibilidade de tratar uma condição grave sem sair de casa. A jornada, porém, ainda passa por regulação de preços, negociações com o SUS e revisões que podem se estender por meses — lembrando que entre a descoberta científica e o alívio humano existe sempre um corredor burocrático a ser percorrido.
- Pacientes com HPN enfrentam anemia crônica, dores severas e risco de morte por trombose, enquanto percorrem em média 101 km — e até 616 km no Norte — para receber infusões em centros especializados.
- A aprovação do fabhalta pela Anvisa rompe um paradigma: pela primeira vez, o tratamento pode ser feito em casa, duas vezes ao dia, sem agulhas e sem deslocamentos extenuantes.
- Antes de chegar às prateleiras, o medicamento precisa ter seu preço definido pela Cmed, e a Novartis ainda não divulgou qualquer estimativa de valor.
- A inclusão no SUS, prevista para ser solicitada no segundo semestre de 2025, enfrentará uma análise de até 270 dias pela Conitec — um horizonte que mantém a incerteza para populações vulneráveis.
- O sistema público ainda não incorporou o ravulizumabe, aprovado anteriormente, sinalizando que a aprovação regulatória não garante acesso imediato pelo sistema de saúde.
A Anvisa aprovou na segunda-feira o fabhalta, desenvolvido pela Novartis, tornando-o o primeiro medicamento oral para a hemoglobinúria paroxística noturna (HPN) — uma doença genética rara em que o sistema imunológico destrói as próprias células vermelhas do sangue. A condição, que atinge cerca de 1,3 pessoa por milhão ao ano no Brasil, provoca anemia crônica, fadiga intensa, dores e complicações potencialmente fatais como tromboses.
Até agora, os únicos tratamentos disponíveis — eculizumabe e ravulizumabe — exigiam infusões intravenosas ou subcutâneas em centros especializados. Um estudo de 2024 revelou o peso dessa logística: pacientes percorriam, em média, 101 km para receber cuidado, distância que chegava a quase 616 km nas regiões Norte do país. O fabhalta muda essa equação ao permitir que o paciente tome o medicamento em casa, duas vezes ao dia, sem depender de estruturas hospitalares.
A aprovação regulatória, contudo, é apenas o primeiro degrau. O preço do medicamento ainda precisa ser fixado pela Cmed antes de qualquer comercialização — e a Novartis não apresentou estimativas. A empresa planeja solicitar a inclusão no SUS no segundo semestre de 2025, pedido que passará por uma análise de até 270 dias pela Conitec.
O cenário é agravado pelo fato de que o ravulizumabe, recomendado favoravelmente pela Conitec no ano passado, ainda não está disponível no sistema público. O Ministério da Saúde segue analisando o protocolo clínico que regulará o acesso a esses tratamentos. Para os pacientes com HPN — para quem o transplante de medula, única opção curativa, é considerado arriscado demais —, o fabhalta representa uma promessa de alívio real. Se e quando essa promessa se tornará acessível depende de decisões que ainda estão por vir.
Brazil's health regulator gave the green light on Monday to fabhalta, an oral medication that represents the first pill-based treatment for hemoglobinúria paroxística noturna—a rare genetic blood disorder that attacks the body's own red blood cells. The approval by Anvisa, the country's pharmaceutical watchdog, marks a shift in how patients with this condition might manage their disease, though significant hurdles remain before the drug reaches those who need it.
HPN, as the condition is known, strikes roughly 1.3 people per million annually in Brazil. It begins with a genetic mutation present from birth that leaves red blood cells without a protective outer layer. This makes them vulnerable to the complement system, part of the immune machinery that normally defends against infection. In people with HPN, this system turns against the body's own cells, destroying them in the bloodstream—a process called hemolysis. The result is chronic anemia, severe fatigue, pain, and potentially fatal complications like blood clots, which remain the leading cause of death among these patients.
Until now, the only treatments available required needles: injections into veins or the fatty tissue beneath the skin, administered at specialized medical centers. Two such medications—eculizumabe and ravulizumabe—are already approved in Brazil. Eculizumabe requires infusions every two weeks; ravulizumabe every two months. For patients, this means regular trips to treatment facilities. A study published in late 2024 revealed the geographic burden: patients traveled an average of 101 kilometers to reach care. In Brazil's northern regions, that distance ballooned to nearly 616 kilometers. Even in the relatively well-served southeast, patients averaged 37 kilometers each way.
Fabhalta, developed by Novartis, works by blocking the proteins that drive the complement system's attack on red blood cells. In clinical trials, patients taking the drug twice daily saw their anemia prevented and symptoms controlled. Rodolfo Cançado, a hematologist at Santa Casa de São Paulo, emphasized that beyond matching the effectiveness of existing treatments, the oral formulation offers something more fundamental: patients would no longer need to travel to specialized centers for infusions. They could take their medication at home.
But approval from Anvisa is only the first step. Before fabhalta can be sold in Brazil, its price must be set by Cmed, the government body that regulates pharmaceutical pricing. Novartis has not provided an estimate for what that price might be. Only after pricing is finalized can the drug enter the commercial market. The company plans to request inclusion in the SUS, Brazil's public health system, in the second half of 2025. That request will then face a 180-day review by Conitec, the commission that evaluates new technologies for public coverage—a timeline that can be extended another 90 days if circumstances warrant.
The approval arrives as the health system grapples with expanding access to HPN treatment more broadly. Ravulizumabe, the other newer option, received a favorable recommendation from Conitec last year but remains unavailable in the public system. The Ministry of Health says it is still analyzing technical data and public consultation feedback on the clinical protocol that governs HPN diagnosis and care. That protocol will determine how and where these medications fit into the public system's offerings.
For patients living with HPN, the path forward remains uncertain. The only truly curative option—bone marrow transplant—carries such high risk and mortality that doctors do not recommend it. What fabhalta promises is not a cure but relief: the chance to manage a serious, life-threatening condition without the burden of constant travel, without the needle, without the dependence on distant medical centers. Whether that promise reaches patients depends on decisions still to come.
Citações Notáveis
The new medication allows patients to take a pill twice daily at home rather than travel to a center for intravenous infusion, and it is more effective at controlling anemia.— Rodolfo Cançado, hematologist at Santa Casa de São Paulo
In HPN, the immune system destroys defective red blood cells in the bloodstream, causing anemia, severe fatigue, pain, and potentially fatal complications like thrombosis.— Phillip Scheinberg, chief of hematology at Beneficência Portuguesa de São Paulo
A Conversa do Hearth Outra perspectiva sobre a história
Why does it matter that this is an oral medication rather than an injection?
Because for a patient with a rare disease, the difference between a pill twice a day and a trip to a specialized center every two weeks is the difference between having a life and managing a disease. Some patients were traveling 600 kilometers for treatment.
But the drug isn't available yet, right? What's actually changed today?
Anvisa said yes—it's safe and effective. But now it has to be priced, and then the public health system has to decide whether to cover it. That could take months or longer.
So who benefits first—private patients or public system patients?
Whoever can afford it once the price is set. The public system won't have it until Conitec approves it, which won't happen until at least the second half of this year, and that's if everything moves on schedule.
What makes this disease so hard to live with?
Your own immune system is destroying your red blood cells. You're constantly exhausted, in pain, and at risk of blood clots that can kill you. And you have to go to a hospital to get the only treatment that works.
Are there other treatments already available through the public system?
One—eculizumabe. It works, but you need infusions every two weeks. The newer option, ravulizumabe, was recommended for coverage but still isn't available. So for most patients, it's still the same old burden.
What does Novartis get out of this?
A market. HPN is rare, but it's serious and lifelong. A patient on this drug is a patient for life.