uniQure schedules critical FDA meeting to revisit request for approval of Huntington’s disease drug

 

uniQure announced on January 9 that it has scheduled a high-priority meeting with the U.S. Food and Drug Administration (FDA) to revisit its application to seek approval of its gene therapy drug for Huntington’s disease, after the agency had rolled back its permission.

 

As recounted here, uniQure and others in the frustrated biotech sector believe that the FDA has become dysfunctional under the Trump administration (click here and here for details).

 

In September, uniQure announced that its drug, AMT-130, had slowed the progression of HD by 75 percent over three years – a historic first. However, in November uniQure announced that the FDA had abruptly switched gears regarding its agreed-to plan for considering AMT-130.

 

In its January 9 press release, uniQure stated that it had scheduled a so-called Type A meeting with the FDA “to support accelerated approval of AMT-130.” It did not disclose the date of the meeting.

 

“We look forward to a constructive discussion with the FDA as we work toward a timely resolution regarding an accelerated approval pathway for AMT-130,” stated uniQure CEO Matt Kapusta. “The Huntington’s disease community, including patients and clinicians, has emphasized the profound unmet medical need and the importance of timely access to potentially disease-modifying therapies such as AMT-130. We remain deeply committed to patient access while continuing to collaborate closely with the FDA.”

 

Seeking to resolve the dispute over AMT-130

 

As noted by the scientist-written website HDBuzz, Type A FDA meetings “occupy a special category.”

 

“They’re reserved specifically for situations where a development program has stalled, like when there’s a clinical hold, major safety concerns, or formal dispute that need to be addressed before a drug can move forward,” HDBuzz explained.

 

Type A meetings are scheduled within 30 days of a request, HDBuzz stated, adding that a Type A meeting can help to “resolve disputes” about clinical trial design or regulatory decisions, “chart a path forward,” and “clarify expectations” so that firms know precisely what the FDA requires. (Click here for the FDA’s official explanation of its meeting types.)

 

Based on this timeline, news from the meeting could be reported in late January or February. The uniQure release stated that the company would provide an update after receiving official minutes from the FDA.

 

Pressing our leaders

 

As of January 12, nearly 47,000 individuals had signed the two petitions from the HD community urging the FDA to expedite approval of AMT-130.

 

The Huntington’s Disease Society ofAmerica also has urged advocates to contact U.S. representatives and senators to “back fair review of AMT-130” by the FDA (click here to write your representatives).

 

In addition to signing one of the petitions and writing to Congress, I will call my Senators and Representative to explain the importance of this cause for the HD community and beyond.

 

I urge everyone to press our political leaders on AMT-130.

 

As I wrote last month, “the FDA should be on the right side of history in this urgent fight to end devastating diseases.”

 

Huntington's Disease Society of America alert: urge Congress to back fair review of AMT-130 (screenshot by Gene Veritas, aka Kenneth P. Serbin)

At-risk Angels pitcher Joe Smith at Huntington’s fundraiser: ‘I’d give every dime I have for a cure’

No one person is the face of Huntington’s disease the way ALS is associated with Lou Gehrig or Parkinson’s disease is linked with Michael J. Fox. But HD touches many lives, including some we know from major league sports.

Choking back tears, 31-year-old Los Angeles Angels baseball pitcher Joe Smith remembered the phone call three years ago from his father back in his native Ohio that changed his life forever: his mother had been diagnosed with Huntington’s disease.

“Unfortunately, I got a call driving home from our spring training site in Goodyear, Arizona, from my dad,” Joe told an audience of over 400 people at a San Diego fundraiser on October 10. “He told me: mom had HD.”

Then Joe recalled when his mother Lee came on the phone on that day in February 2012, not long after she had received her genetic test results confirming she had HD.

“I’ll never forget the sound of her voice,” he said. “It was just empty. It was the worst. I never heard anything like it. That stayed with me for a long time, that sound, when she said, ‘Hi, Joseph,’ but the way she said it […] was different. And it hurt. It still does, obviously. This time, when she got the news, I still didn’t know a whole lot about HD. But obviously, when you get off the phone with the parents and got a 30-minute drive, there’s a lot of thinking that goes on.”

He’s done a lot of thinking – and action – since then.

For his efforts to raise awareness and funds for the HD cause, Joe received the Guthrie Award of the Huntington’s Disease Society of America (HDSA) at the San Diego chapter’s 15th Annual Celebration of Hope Gala, held this year at the spectacular coastal residence of Craig and Rebecca Irving. Craig is a businessman and philanthropist.

Above, Joe Smith and mother Lee (photo by Gene Veritas, aka Kenneth P. Serbin). Below, Celebration of Hope Gala attendees mingling before the start of dinner and the formal program (photo by Mike Nowak).

Staring HD in the face

Joe remembered his family’s four-hour drives from southern Ohio to Cleveland to visit Lee’s mother, who, the family knew, suffered from HD. Seeing his grandmother’s progressively worsening symptoms at each visit left Joe sad and concerned as a child and teenager.

“I think that’s the scariest part,” Joe continued. “It’s one thing, I think, to go through life, or to have something that not necessarily you don’t know about, but […] my mom took care of my grandma, she went to doctor visits with my grandma, she was on the phone all the time with my grandma.

“When you know the road you’re heading down, and right now there’s nothing that you can do about it, you’re just going. You’re hoping there’s light. You got hope. You got faith. But at the end of the day, right now, there’s no cure.”

Lee did not speak at the gala but talked about her symptoms in a brief video shown to the audience.

“She stares it right in the face every day,” Joe said, referring several times to his mother’s fortitude.

Joe ended his speech with a call to boost fundraising for HD research.

“I’d give every dime I have if they had a cure today,” he declared.

You can watch Joe’s speech in the video below. View other videos of the event by clicking here.

Los Angeles Angels' Joe Smith: 'I'd Give Every Dime I Have for a Cure for Huntington's Disease' from Gene Veritas on Vimeo.

Taking public action

As the children of an HD-affected parent, both Joe and his 29-year-old sister Megan Nein have a 50-50 chance of inheriting the genetic defect.

Joe has previously talked to the press about his fears of living at risk.

“My sister has three kids and she hasn't been tested,” Joe said last March. “I got married recently, and I'll get tested before we have kids.”

He didn’t speak directly about his fears at the gala, but they were palpable throughout his speech.

Both Joe's and HDSA CEO Louise Vetter’s comments once again demonstrated how HD can devastate the extended family because of its genetic cause and difficult caregiving burden.

“It’s not easy to come out and say you’re from an HD family,” Vetter said in introducing Joe. “If one of your parents has it, you don’t know if you have it or not. So it takes a lot of courage to face your future.”

Recalling Woody Guthrie’s widow Marjorie’s founding of HDSA in 1967, the organization recognized Joe with the Guthrie Award because of his “bravery” in confronting HD, she said.

Too often conversations about HD take place “behind closed doors,” Vetter observed.

The Smith family has “made it public and they’ve created a call to action,” she explained.

HDSA CEO Louise Vetter (photo by Mike Nowak)

The community emerging as its own spokesperson

For older generations of Americans, songwriter-activist Guthrie symbolized HD.

In recent decades, with younger generations unacquainted with Guthrie, many in the Huntington’s community have attributed the lack of awareness about HD – ironically one of the most common of the rare diseases – to the lack of a celebrity such as Michael J. Fox in the sphere of Parkinson’s disease.

Without national opinion polling on HD, we can’t really know if this is the case.

What’s important is that more HD family members are telling their stories publicly than ever before, and HD is gaining exposure.

Like 33-year-old filmmaker-actress Marianna Palka, who revealed her HD genetic test results in an HBO film that premiered in June, Joe is emerging as a key new spokesperson for the HD cause.

Two other successful athletes – former Olympic rower Sarah Winckless and former National Hockey League player Jake Dowell – have shared their HD stories.

In June, another, award-winning film, the documentary The Huntington’s Disease Project: Removing the Mask, was released.

Networking for the cause

Joe’s advocacy is helped by the fact that professional baseball maintains a huge fan base.

Joe and his family have started a foundation, Help Cure HD, to raise money for research on deep brain stimulation (DBS) as a potential treatment for HD. (For years doctors have used DBS to treat Parkinson’s disease.) So far Help Cure HD has raised nearly $400,000.

Joe’s wife is Allie LaForce, a TV reporter for CBS Sports. In January, Allie did a feature on Bill Johnston, the public relations director of the National Football League’s San Diego Chargers, and his fight against HD. Bill’s wife Ramona, who has HD, now lives in a nursing home.

The main mover behind the smartly produced HDSA-San Diego galas, Bill has helped raise several million dollars for HDSA through those events and numerous others.

Bill uses his contacts in the upper echelons of business and pro sports to invite speakers like Joe and garner corporate sponsors such as the B. H. Gold Insurance Agency. HDSA also honored B.H. Gold President Bill Habeger with the Guthrie Award for his support of the cause.

“May these galas soon be victory galas,” Habeger told the audience.

HDSA-San Diego President Burt Brigida (left), B.H. Gold President Bill Habeger, HDSA CEO Vetter, and HDSA-San Diego immediate past president George Essig (photo by Mike Nowak)

From the heart, emboldening our community

As they arrived at the gala, I introduced myself to Joe, Lee, Joe’s father Mike, and Tim Mead, the Angels’ vice president for communications.

I told Joe that I was also from Ohio, that my mother had died of HD, and that I carried the genetic defect. I told him that he could rely on the San Diego chapter, HDSA, and me for anything he and his family might need in the struggle against HD.

Lee and I shared a few words about our common Cleveland connection. When she mentioned that she was 56, I said I was right there with her at 55.

At 56, my own mother had the involuntary movements typical in HD and was starting to lose her ability to reason.

Joe (left), Lee, and Mike Smith with Tim Mead, vice president for communications, Los Angeles Angels (photo by Gene Veritas)

I thought of how lucky I was to remain asymptomatic and participate fully in the gala.

After his speech, Joe asked my opinion.

“You hit it right on the mark,” I said. “You spoke from the heart.”

Later, just before the end of the gala, I spoke again with Joe. “We are brothers in this cause,” I said, putting my arm around his shoulder.

Joe raised awareness and money, but most importantly he has emboldened our fellow HD brothers and sisters to join the fight. Having every dime in the world won't bring treatments unless we have enough participants in the all-crucial research studies and clinical trials.

Joe Smith (left) and Gene Veritas (photo by Mike Nowak). Watch more videos of the gala by clicking here.