A banner on the tallest building of the Vall d'Hebron hospital complex in Barcelona reads: "Estimate life." Eight-year-old Nadia, who has just come out of a routine check-up at this center's maternal and child hospital, also loves life. They have pricked her finger, but they have put a colorful band-aid on her. From her Two teeth have fallen out, but Pérez the mouse has brought her a beautiful crying baby.
Nadia, like her parents, always smiles, even though life has put many obstacles in her way. She was born with achondroplasia, a genetic mutation that alters the normal growth of the limbs and causes an abnormal short stature, in addition to many limitations and a host of problems: muscular hypotonia, macrocephaly... She also has refractory epilepsy, among other additional ailments. And despite everything, she always smiles.
Growing a few more centimeters is not an aesthetic issue, but rather one of quality of life: the length of the arms of some boys and girls, for example, does not allow them to reach certain parts of their body and be autonomous in their hygiene. Their families could resort to surgical lengthening, but it is a very invasive, painful, complicated method and not without clinical risks... And then Voxzogo arrived.
This pioneering medicine helps the bones of children with achondroplasia to grow. There are photos that show spectacular results in patients with just a few months of treatment. The European Medicines Agency approved the drug in 2021 and its Spanish counterpart did so on January 20. Since February 1, the product has been financed by the National Health System (SNS).
In hospitals in communities such as Murcia, Cantabria or Asturias, among others, it is already distributed. In Catalonia it has not yet arrived, despite the fact that it is already authorized and paid for. The Catsalut or Servei Català de la Salut, the body in charge of the public healthcare system of the State in Catalonia, admits that Voxzogo "is available to be dispensed at the SNS from February 1".
This implies, adds Catsalut, that the product "is also financed in Catalonia from that same date." However, to the despair of 13 Catalan families (14, according to other sources) it is still not distributed in Catalan hospitals. Some parents consider registering in another community "to have access there to what we don't have here." Others do not rule out appealing to justice to expedite the administration's response.
Voxzogo is given as daily injections up to 16 years of age, after which the bony growth plates close and it is no longer useful. “We are wasting precious time that we will never be able to recover”, explain Nadia's parents. In Catalonia there are more than a dozen boys and girls like her, with achondroplasia and under 16 years of age. This haste, however, does not affect the protocols of Catsalut.
“One of our priorities –says this organization– is that all possible patients in Catalonia receive the medicine in fair conditions and that the best possible use is made of them”. This and all "innovative drugs with a relevant clinical impact are evaluated by the harmonization program of Catalonia." The management of the Servei Català de la Salut affirms that "this assessment is very advanced".
But a day passes. And other. And other. According to one of the latest official communications, the meetings of the evaluation committee have already begun, which is studying "possible reference centers for pharmacological monitoring", among other issues prior to "the ordinary use of the product". The procedure, Catsalut has advanced, will just be defined "in a last meeting on April 5".
Desperate parents, who have the support of the Alpe Achondroplasia Foundation and the Spanish Federation for Rare Diseases, wonder if “it couldn't be sooner”. Days do not always last 24 hours. A day is an eternity for them. Every day without the injection is one day less for children who urgently need to improve the alignment of their spine, legs, arms and hands. And, above all, improve their quality of life.
