NEW HARTFORD -- The family pleading with the pharmaceutical giant GlaxoSmithKline to allow them access to the seizure medication Potiga is making progress.
One-year-old Christian Mumm and his twin brother are the youngest of five children. Christian was born with a spontaneous mutation to the gene KCNQ2 which means he has frequent and severe seizures. His condition has left him with a terminal prognosis.
His parents say Potiga is one of a roughly half a dozen medications that Christian’s doctors have tried that minimize his clusters of seizures and allow him better quality of life.
Since GSK pulled Potiga off the market in late June due to a lack of demand, the Mumm’s have asked for the drug to be available to them on a “compassion use” basis. In recent weeks their request has gained some ground.
“Congressman Larson became so dedicated to Christian and joined team Christian and really took us to the next step with opening communications with GlaxoSmithKline,” Christian’s mom, Erica Mumm, said. She said she, her husband, Christian’s doctors, and GSK all spoke recently over the phone.
“In this conference call it was collaborative, we do feel positive about it,” Mumm said. She went on to say, “We had a good conversation, I personally spoke on the dire need that Christian has for Potiga and I think that that’s something that they didn’t realize or don’t hear enough of.”
Mumm also said that she had not been given a final answer as whether the medication will in fact be made available to them, and if so, for how long.
FOX61 were in contact with GSK representatives on the matter Monday who said they do believe they have a solution to the issue and are close to an answer. They also stated the following:
“We are aware of two children with the rare genetic condition, KCNQ2 Encephalopathy who are currently being treated off label with Potiga. We have proactively reached out to these families and their treating physicians' in order to evaluate potential pathways to making the product available for these children. One of the families is the Mumm family. We spoke with both doctors treating these patients, and at their request have now discussed with the FDA a path to provide these two children with access to the medicine from within our existing remaining supply. We are absolutely committed to doing everything we can to respond to these patients needs and their doctors' requests.”
Larson, who has been pushing for the Mumm’s voices to be heard on the issue, applauded GSK’s efforts and praised the Mumm’s for the persistence. He told FOX61, however, Christian’s story is one of a much larger picture which highlights the pull between for-profit drug companies and moral obligations to patients.
"When you look at the whole issue of orphan drugs and when you look at other diseases or genetic disorders in this case, we've got to find a way that we can come to logical conclusions,” Larson said.
For Christian, the clock is ticking as the Mumm’s have a roughly three month supply of Potiga left for their son, but say this is a fight they will not give up on.
“We will continue to pursue different avenues, different pathways 2353 until a resolution is found so that he can have the best quality of life,” Mumm said.
Christian is currently being cared for at the Hospital for Special Care.