TORONTO -- It鈥檚 only Eva Batista鈥檚 first birthday but for her parents, it feels like time is running out.
By Eva鈥檚 next birthday, she may have already aged out of eligibility for a life-saving drug that Ricardo and Jessica Batista are racing against the clock to pay for.
Their one-year-old daughter has spinal muscular atrophy (SMA) and needs to receive a single, one-time dose of Zolgensma before she turns two, or reaches an unspecified weight, for the drug to be effective. The rare neuromuscular disease causes muscle weakness and atrophy, impacting simple actions like Eva鈥檚 breathing, swallowing and the ability to hold her head up. Most SMA patients like Eva, who has the worst form of the disease, don鈥檛 live past the age of two.
鈥淪ome families never got the chance to celebrate their children鈥檚 first birthday with this disease. To have her here is a blessing,鈥� her mother told CTVNews.ca over the phone late last month.
The Zolgensma gene therapy is also known as AVXS-101 and is produced by Swiss drug company Novartis, has yet to be approved by Health Canada. The drug, which is more effective the earlier it is given, replaces a defective gene that causes SMA and has been approved for use in the U.S., Japan and the European Union.
Novartis recently filed for approval in Canada earlier this year and was granted 鈥淧riority Review status,鈥� a spokesperson told CTVNews.ca in an email. 鈥淲e are committed to working collaboratively with regulatory, pricing and reimbursement authorities across Canada in providing eligible patient access to AVXS-101,鈥� they wrote. 鈥淲e are eager to finalize discussions and partner on solutions as quickly as possible.鈥�
If approved by Health Canada, the drug would move into a second longer phase when provincial governments would assess funding for the treatment, a process that might be too lengthy for Eva.
鈥淯nless they make an exception for her case 鈥� by the time they resolve everything it will be too late for her. That鈥檚 why we continue with the fundraising,鈥� said Ricardo over the phone. 鈥淲e can鈥檛 wait to hope that she鈥檒l make it before the [two-year] cut-off time. If she doesn鈥檛, she鈥檚 out of luck. We鈥檙e not leaving it to that in hopes that they鈥檒l have consideration for her, because at the moment there hasn鈥檛 been any.鈥�
BUYING A BETTER LIFE
To access Zolgensma independently costs a fortune. The gene therapy is considered the , with a price tag of US$2.125 million. It would cost the Batista family upwards of CAD$2.8 million to buy it for Eva and have it administered in Canada. They have raised about $2.2 million and are aiming to raise $800,000 more.
But the COVID-19 pandemic has dampened their funding efforts. Most of the money raised came from large in-person events. A car show and dinner for hundreds of people pulled in more than $100,000 each in December and January alone.
An called 鈥淔orEva Strong鈥� has brought in about $955,000 over the course of 10 months.
Eva is not alone in Canada. The family of an named Kaysen have been fundraising for Zolgensma all summer and have raised just over $1.1 million of a $3-million goal . Kaysen turned two in July, but the family was told that there is 鈥渨iggle room鈥� on when the drug can be administered. In Vancouver, the parents of four-month-old Lucy raised more than $2.4 million in a few short months and recently announced the girl would officially be getting the drug.
BETTING ON THE LOTTERY
Other families around the world have been lucky to receive the drug for free through a lottery system offered by Novartis. In January, the drug company launched a 鈥渂i-weekly random allocation process鈥� to provide 100 doses of Zolgensma worldwide by the end of the year.
The Batistas know of at least one Canadian family whose daughter received the drug through the Novartis lottery in April at Toronto鈥檚 Hospital for Sick Children in April. The results for that child were 鈥渁mazing,鈥� said Jessica.
鈥淏efore, she wasn鈥檛 even able to really hold her head up, she barely moved her arms, she would not move her legs at all,鈥� she said. Within the first month or two after receiving Zolgensma, the girl gained strength, could hold up her head and was moving and lifting her legs.
鈥淭he results were huge and you could see it right away,鈥� said Jessica.
If they win the lottery, the Batistas have received special authorization from the Canadian government to bypass the approval process and have the drug administered, but they aren鈥檛 betting on it. If Zolgensma isn鈥檛 approved before Eva turns two -- or they don鈥檛 win the lottery, or they can鈥檛 raise enough money -- Eva will remain on an alternative life-long therapy called Spinraza. That drug is approved in Canada and costs over $700,000 in the first year and more than $350,000 for subsequent years. While Spinraza has helped with strength and movement for Eva, who was diagnosed at just seven weeks old, her respiratory system hasn鈥檛 improved.
All signs suggest that Zolgensma would be a life-changing treatment for the Batistas, though one-year-old Eva doesn鈥檛 know anything is wrong.
鈥淪ometimes we look at her and it kills us inside that she鈥檚 so happy and she has no idea,鈥� Ricardo said during an emotional interview on CTV鈥檚 Your Morning earlier this year.
鈥淪he鈥檚 a beautiful little girl that always has a smile on her face.鈥�
Her first birthday celebration Tuesday would be no exception, even if the festivities couldn鈥檛 be quite what Eva鈥檚 parents had hoped prior to the COVID-19 pandemic. But they鈥檒l honour her strength even if it鈥檚 just the three of them.
鈥淪he deserves it for what she鈥檚 accomplished,鈥� said Ricardo. 鈥淎t the end of the day, she鈥檚 the one that fights through it. Not us.鈥�
