May 24, 2019 at 12:35 pm #19027Michael MoraleKeymaster
We just received notification that the FDA has officially approved ZOLGENSMA as the 2nd FDA approved treatment for Spinal Muscular Atrophy https://smanewstoday.com/2019/05/24/fda-approves-zolgensma-gene-therapy-newborns-toddlers-with-any-sma-type/. We are still in the process of learning more information, but this is of course great news! Please follow our main site for more information and further updates.
What are your thoughts? We’d love to hear everyone’s perspectives on this news. Feel free to post questions as well.
May 24, 2019 at 12:50 pm #19033AnonymousInactive
Read SMA News Today coverage of the approval here.
We’d love to hear from you! Give us your feedback on what the FDA approval of ZOLGENSMA means for the SMA community.
May 24, 2019 at 2:48 pm #19043Kevin SchaeferKeymaster
And yes, whether you are someone who has SMA, or a parent/caregiver, we would love to hear from you. Please let us know your reactions, questions, etc.
May 24, 2019 at 3:51 pm #19048
I was actually surprised, in a good way, at the price. I was expecting it to be much closer to the $4-5 million range that had been first suggested. At $425,000 per year for 5 years, it’s close to Spinraza’s price when you factor in the costs of the procedures every 4 months, and then there’s nothing beyond that which makes it significantly more cost effective over the long run.
May 24, 2019 at 6:34 pm #19049Ryan BerharMember
May 24, 2019 at 9:19 pm #19051Halsey BlocherParticipant
This will be such a blessing! At one point in time, I never thought I’d see the day that there was an SMA treatment, let alone two! Hope to see this studied for adults in the near future!
May 25, 2019 at 8:22 am #19052DeAnn RKeymaster
It almost seems like something from a science fiction novel. What an amazing advancement in science. It’ll be exciting to see where they go from here. As Lupa said although the cost is high, it’s surprisingly lower than anticipated. I have to wonder if this competition will put pressure on Biogen to reduce the cost of Spinraza, or if Zolgensma will be the treatment of choice for children under 2 and Spinraza will be for everyone else. Also hoping access to this treatment will be smooth for families in need.
May 26, 2019 at 11:03 am #19057Grace FrankParticipant
Hi all! Please do share your thoughts about this first gene therapy for SMA.
Are you hoping to use it soon? Are you talking with your doctors about Zolgensma?
Do you think it will be approved for older patients with types 2 and 3 this year? Next year?
Or, because it’s only now only being tested in type 2 children up to age 5 (STRONG trial), do you think the FDA might be much slower to act and approve the intrathecal (spinal canal) delivery that’s necessary for teens and adults with types 2 and 3?
Does its price ($2.125 million for one-time use) worry you, or has this become a “reasonable” price in the rare disease world?
You’re the experts here. What do you think?
May 26, 2019 at 3:10 pm #19063
“Do you think it will be approved for older patients with types 2 and 3 this year? Next year?”
Because it’s supposed to be a one-time treatment, I think they’re going to need longer term data on older patients to make sure it’s still effective years down the road so I don’t think it’ll be approved any time soon.
“Does its price ($2.125 million for one-time use) worry you, or has this become a “reasonable” price in the rare disease world?”
The price is ridiculous, but these companies can get away with it because insurance companies are going to pay any price so they’re not accused of letting children die. These companies are putting up ransoms on children’s lives and there’s no choice but to pay them.
As I read elsewhere, you don’t normally pay for drugs or treatments like they’re pricing these SMA treatments. The one example I saw was if you need your appendix out. If you don’t, you’ll die, but you don’t get charged for the surgery based of the extra number of years you’ll now live. The surgery has a fixed cost based on the time and effort required to do it. You don’t get a bill saying you owe $2 million because otherwise you’d be dead, but that’s how they’re pricing these SMA and other rare drug treatments.
May 26, 2019 at 5:19 pm #19065
From https://www.cnbc.com/2019/05/24/fda-approves-novartis-2-million-spinal-muscular-atrophy-gene-therapy.html: “Insurers were going to cover Zolgensma no matter the price, and Novartis has spoken publicly about considering prices that approached $5 million,” ICER President Steven Pearson said in a statement.
May 28, 2019 at 8:30 am #19078Grace FrankParticipant
Thanks for sharing your thoughts, Lupa!
June 4, 2019 at 3:00 am #19200Adela MohanuParticipant
I was wondering if anyone started the process of receiving Zolgensma. What are the steps? Are the doctors willing to prescribe it? Do the companies accept the prohibitive price?
It would be nice to find out about the first patient who received it not in a trial, but through a regular insurance.
We are an Italy-based family with a 15mo son who is sma1 and I think that the next weeks will also give a trend for Europe. While I hope it will be soon approved, in Europe we have different kind of insurance policies and basically the governements have to be willing to sustain the costs. I fear that they will look at any rejection in US and use it as an argument.
Joyfull days to you all!
June 4, 2019 at 12:53 pm #19213Kevin SchaeferKeymaster
So because the approval just happened, it’ll be some time before we talk with anyone who receives the treatment. Our news department did recently speak with a parent, whose son took part in the zolgensma clinical trial. We’ll keep you posted. In the meantime, follow our main website at http://www.smanewstoday.com
Thanks for your input!
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