With the ‘worst’ FDA head out and Huntington’s disease advocates pressing for bipartisan reform, uniQure announces new plan to seek gene therapy approval

  

On June 17, uniQure announced a new plan to apply to the U.S. Food and Drug Administration (FDA) to seek approval of its gene therapy for Huntington’s disease  – a dramatic shift after the agency had last year blocked the drug despite results showing, for the first time, that HD progression could be slowed.

 

The announcement comes in the wake of the May 12 resignation of the head of the crisis-ridden FDA, which has clashed with uniQure.

 

In a press release, uniQure reported that, at a recent meeting with the FDA, the agency had accepted that the three-year analysis of the drug, AMT-130, which the company has presented as demonstrating efficacy against HD, can serve as the “primary basis” for a drug approval application.

 

uniQure aims to apply in the third quarter of this year. In alignment with the FDA, the company will also conduct a “confirmatory study” to further test the efficacy of AMT-130. According to early-stage clinical trial results reported by uniQure, AMT-130 had demonstrated a 75 percent slowing in HD over a three-year period – a historic achievement.

 

For the confirmatory study, uniQure will work with the FDA to determine how to evaluate a “concurrent control,” that is, a placebo or comparator to measure drug efficacy in those not receiving the drug. According to the press release, this study would not include a sham surgery as a placebo – a requirement introduced in March by the FDA but considered by many to be unethical because of the already harmful symptoms of HD.

 

According to the press release, the FDA has agreed that uniQure can, for a comparator, still use the patient information from the important Enroll-HD database of affected individuals – a major point of contention in the FDA’s surprise reversal of its promises regarding the study of AMT-130.

 

“Today's announcement reflects the outcome we have worked toward throughout our continued regulatory engagement with FDA, and we are deeply grateful for FDA’s genuine commitment to addressing the unmet need of Americans living with Huntington’s disease,” Matt Kapusta, uniQure CEO, stated in the release. “The FDA has agreed that our current clinical data can support a near-term BLA [biologics license application] submission and has committed to work expeditiously with us to align on the design of the required confirmatory study.”

 

‘A meaningful step forward’

 

The new understanding with the FDA represents a significant shift, because, after previously clashing with uniQure, the FDA recommended in March that the firm conduct a full-blown Phase III clinical trial, including the use of a sham surgery.

 

“Today’s announcement from uniQure represents an encouraging and meaningful step forward for the Huntington’s disease community,” Amy Gray, president and CEO of the Huntington’s Disease Society of America (HDSA), stated in a press release. “I applaud the leadership at the FDA for allowing uniQure to take this important step forward in the development of its investigational treatment for Huntington’s disease.

 

Gray added that “this progress did not happen in isolation.” Following “regulatory hurdles,” the HD community “united like never before.” HD advocates delivered to the FDA two petitions with more than 48,000 signatures in favor of AMT-130. According to Gray, more than 11,000 messages to Congress, participation in legislative meetings, and sharing of personal stories added to the impact.

 

“I am deeply grateful to the Members of Congress who stood with Huntington’s disease patients and families during this effort,” Gray said. “Their willingness to engage with the FDA, ask important questions, and advocate for a regulatory framework that reflects the realities of rare disease research helped ensure that the voices of our community were heard.”

A whirlwind of developments

 

The uniQure announcement about AMT-130 came in at the end of a whirlwind of recent developments. The HD community has regrouped in support of improved drug discovery policy at the crisis-ridden FDA.

 

On April 30, uniQure took the case for its drug to the United Kingdom’s Medicines and Healthcare products Regulatory Agency (MHRA) as a first step to seek approval in that country. The company plans to submit an application in the third quarter of 2026.

 

On May 12 FDA Commissioner Marty Makary, a Trump administration appointee, resigned after what STAT considered to be the “worst” leadership of the agency in 25 years because of “a fundamental lack of understanding of the nature of the role, of the functions of his agency, and of the needs of the employees who worked for him.” His departure prompted calls for rebuilding public trust in, and better leadership at, the FDA (click here and here to read more).

 

On June 1, the FDA announced that Acting Commissioner Kyle Diamantas would meet with rare disease group leaders to seek to steady operations and mend fences with disease groups. That meeting took place on June 3.

 

Jeff Allen, CEO of Friends of Cancer Research, described the encounter as a “breath of fresh air.”

 

The FDA did not invite HDSA and other HD advocacy organizations to join the meeting. The FDA declined to answer questions. As of publication time, a message left at the National Organization for Rare Disorders seeking comment had not been returned.

 

A key briefing on rare diseases

 

However, on June 2, HDSA CEO Gray moderated a panel of five rare disease community representatives at a bipartisan congressional townhall briefing in Washington, D.C., titled “The Pathway to Cures and Treatments for Rare Diseases.”

 

“Rare disease families need a clear and sustainable pathway to research, treatments, and care,” said Gray in an HDSA press release about the event, available on YouTube. “This briefing is an important opportunity to bring patient advocacy organizations, scientific leaders, policy experts, and lawmakers together to discuss how we can advance meaningful progress for families impacted by rare diseases.”

 

With more than 40 people in attendance, the meeting highlighted the suffering of people with rare diseases, the urgent need for effective treatments, and the need to improve the FDA’s procedures. It included comments from both a Democratic and a Republican member of the House of Representatives.

 

The Pathway to Cures and Treatments for Rare Diseases congressional townhall briefing at the Rayburn House Office Building, Washington, D.C., June 2. From left to right, Kathryn Bryant Knudon, The Speak Foundation; Emily Gantman, Ph.D., CHDI Foundation; Lauren Moore, Ph.D., National Ataxia Foundation; Monet Stanford, PharmD, Washington Analysis; Tamara Maiuri, Ph.D., HDSA; Amy Gray, HDSA; and Rep. Jake Auchincloss (screenshot by Gene Veritas, aka Kenneth P. Serbin)

 

Bringing ‘smart’ leadership to the FDA

 

Rep. Jake Auchincloss, a Democrat from the greater Boston area and a member of a family of physician-scientists, said that he was “passionate” about the issue of rare disease drug development. He is on the Subcommittee on Health of the Committee of Energy and Commerce.

 

The subcommittee oversees the FDA. Auchincloss’s focus includes clinical trial reform, which the panelists agreed was important for rare disease drug development.

 

“While the science has never been better in trying to unlock those answers, the politics has never been worse,” Auchincloss said at the briefing.

 

Auchincloss spoke of the need for change on three fronts involving science and research: to bring U.S. research spending back to its previously high levels; to bring “smart” leadership back to the FDA; and to require insurance companies to cover the many new rare disease therapies on the horizon.

 

Auchincloss added that the “most urgent issue now” is to stop the Trump administration’s “unprecedented” cutting of National Institutes of Health grants without the time-honored standard peer review.

 

‘Cut the red tape’

 

Rep. Morgan Griffith, a Republican who represents far western Virginia, chairs the Subcommittee on Health. He supports the cause of the ALS (amyotrophic lateral sclerosis) community and pediatric cancer. He spoke about how people with rare diseases in remote areas have difficulty accessing clinical trials in urban areas.

 

Griffith also spoke movingly of the two HD families he has gotten to know.

 

He agreed with Auchincloss’s approach regarding the FDA and clinical trial reform. The two speak regularly on these issues.

 

“We have to figure out a better way to do it,” Griffith said. “It needs to be bipartisan.”

 

Auchincloss is not seeking more money from the government but simply to “cut through the red tape” regarding drug development, Griffith said, adding that people with rare diseases should not be required to “jump through the same hoops” as those participating in clinical trials for less difficult conditions such as nausea.

 

‘We were finally heard’

 

On September 17 and 18, news about AMT-130 dominated bioscience headlines after the uniQure announcement – and provided a needed boost to the HD community.

 

“I’m literally crying right now,” Lauren Holder, a Help4HD International Advocate and, like me, an HD gene carrier, told STAT. “I’m so happy that I don’t even know how to put what I’m feeling into words.”

 

Holder added, “This is the best-case scenario for our community.”

 

“This happened because dedicated patient advocates refused to give up, because this community continued to show up, speak up, and fight, even when it felt like no one was listening,” she said. “Today, it feels like we were finally heard.”

A Senate probe of the FDA and a letter from advocates to Trump could aid in approval of Huntington’s disease gene therapy

  

Two key political developments could aid uniQure’s effort to seek approval of its historically efficacious Huntington’s disease gene therapy, AMT-130, from the U.S. Food and Drug Administration (FDA): a senatorial probe of the FDA and a letter to President Trump from rare-disease advocates.

 

As reported widely, uniQure is preparing for yet another meeting with the FDA to discuss the potential Phase III large-scale clinical trial required by the agency to further test the efficacy of AMT-130. Since its start in 2019, the Phase I/II trial has involved some 45 people. In September 2025, uniQure revealed that 17 of the individuals receiving the highest dose of AMT-130 had a slowing of progression of HD by 75 percent. The FDA, seen by many biotech observers to have become dysfunctional under the Trump administration, then surprisingly reneged on a promise to allow uniQure to apply for approval in 2026.

 

On March 9, Wisconsin Sen. Ron Johnson, a Republican, announced that he had launched an investigation of the FDA because it had rejected drug applications for several rare diseases.

 

Senator Ron Johnson (official congressional photo)

 

Johnson described the FDA’s request for a new AMT-130 trial, which would include a deeply invasive sham surgery for participants not getting the actual drug, as “bureaucratic idiocy.”

 

AMT-130 requires a twelve-hour surgery to inject the drug deep into the brain. The sham surgery would serve as a placebo in the clinical trial.

 

“You’re expecting people to go through sham surgeries where they get holes drilled in their heads?” Johnson said. “That’s just unbelievable.”

 

As part of his probe, Johnson is seeking denial letters from the FDA. Referring to the agency, he said that the “stories are outrageous. It just appears that they’re looking for excuses to say no.”

 

The senator added: “We’re going to find out exactly what issues the FDA listed for their ‘nos’.”

 

A sham surgery could pose ‘significant risk’

 

Johnson’s office did not respond to my request for an interview.

 

Walid Abi-Saab, M.D., uniQure’s chief medical officer, told the key publication Neurology Today that a sham controlled study “could impose significant risks and burden to patients” and that some people may consider it unethical.

 

Dr. Abi-Saab added that volunteers facing the sham surgery could not access other potentially disease-modifying therapies advancing through trials involving several years of observation.

 

Early in the AMT-130 program, uniQure used a sham surgery in several individuals but then stopped precisely because of ethical concerns.

 

Advocacy groups such as the Huntington’s Disease Youth Organization (HDYO) also have cautioned against the sham surgery.

 

In a March 6 e-mail, the organization stated that the “use of a sham neurosurgical procedure – requiring participants to undergo the risks of major surgery to the head including a lengthy time period under anesthesia without the possibility of therapeutic benefit – places an unnecessary burden on individuals already facing a progressive and fatal condition.” HDYO urged the use of “alternative trial designs … while minimizing avoidable risk to participants.”

 

Seeking to restore regulatory clarity

 

According to a report by Reuters, on April 1 the Rare Disease Advocacy, Biotechnology, and Investor Coalition sent a letter to President Trump, U.S. Health Secretary Robert F. Kennedy Jr., Medicare head Mehmet Oz, M.D., and FDA Commissioner Marty Makary, M.D.

 

The letter urged the leaders to restore regulatory clarity at the FDA as it considers a new leader for the agency’s Center for Biologics Evaluation and Research.

 

Headed by Vinay Prasad, M.D., who has resigned for the second time, the Center evaluates gene therapies such as AMT-130. Dr. Prasad’s departure follows a series of controversies involving the review of vaccines, gene therapies, and biotech drugs, including reversals of FDA promises. Biopharma executives, investors, members of Congress and others have criticized his actions.

The coalition includes nearly 100 patient disease advocacy groups, biotech executives, and investors. The letter noted that the Center had become less flexible in overseeing rare disease clinical trials.

 

The group has observed a drop in rare disease investment because of the uncertainties caused by the FDA.

 

“We believe it is of the utmost importance that the FDA chooses [for the Center] a leader who understands the unique challenges of rare disease development and respects and values the views of patients and physicians,” their letter stated.

 

A chance for the FDA ‘to reset’

 

An April 1 report by the news site BioPharmDive echoed the advocates’ critical analysis of the FDA.

 

The report observed that, after a “turbulent year” at the FDA caused by “constant leadership changes” and “erratic decision making,” the agency is as “unpredictable as it’s ever been.”

 

BioPharmDive concluded that Dr. Prasad’s departure by the end of April “could give the FDA a chance to reset.”  Commissioner Makary promised to name a replacement before he leaves.

 

‘We can’t afford delays for rare diseases’

 

On February 26, Florida Sen. Rick Scott, another Republican, chaired a hearing of the Senate Special Committee on Aging, which addressed the FDA’s difficulties.

 

At the hearing, physicians, biotech leaders and rare disease patient advocates criticized the FDA for stifling innovation.

 

The hearing discussed FDA reversals of previous agreements that it had made with drug makers. One advocate pointed out that, from 2024 to 2025, the FDA had 65 percent fewer advisory committee meetings for new drugs and biologics (a medicine derived from a living organism as opposed to a chemical), including for rare diseases.

 

At the hearing, New York Sen. Kirsten Gillibrand, a Democrat, pointed out that about one in ten Americans have a rare disease. She stated that the FDA is “not working how it should be” as laid out by Congress.

 

“This is heartbreaking for patients, and it’s why we can’t afford delays and disruptions in treatment,” Gillibrand observed. “Rare diseases can progress rapidly, cause irreversible harm and, in some cases, premature death.”

FDA recommends a full clinical trial for uniQure Huntington’s disease drug, further delaying potential approval

  

Delaying but not blocking AMT-130, uniQure’s emerging gene therapy to slow Huntington’s disease progression, the U.S. Food and Drug Administration (FDA) “strongly recommended” that the company conduct a full-blown, Phase III clinical trial, rather than assess an application for drug approval based on an earlier trial that showed historic efficacy.

 

The news came in a March 2 uniQure press release, relying on official minutes from the company’s January 30 high-priority meeting with the FDA about the AMT-130 program. The drug has been shown to slow the progression of by HD symptoms by as much as 75 percent.

 

As discussed below, this action reflects the more conservative trend of at least parts of the FDA under the Trump administration.

 

Based on the meeting minutes, the FDA has maintained its new decision that data from the earlier AMT-130 Phase I/II trial, using the global Enroll-HD patient database as a comparison, cannot be used as primary evidence for a drug application, the release stated.

 

The company added: “The FDA strongly recommended uniQure conduct a prospective, randomized, double-blind, sham surgery-controlled study. uniQure intends to continue engaging with the FDA regarding Phase III development considerations and plans to request a […] meeting in the second quarter of 2026 to further discuss potential study design approaches.”

 

A prospective, randomized, double-blind study is a standard clinical trial in which neither patients nor researchers know who receives the drug. A sham (pretend) surgery, which uniQure did on a few clinical trial participants early in the Phase I/II study, will now be required as a placebo, which is standard in most clinical trials. Relying on Enroll-HD data had taken the place of the sham surgery, which uniQure had stopped doing for ethical reasons. Injecting AMT-130 into the brain involves a twelve-plus-hour brain operation.

 

uniQure’s plans

 

“While we did not reach alignment on a submission pathway based on the Phase I/II data, we believe the totality and durability of our data warrant continued substantive dialogue” with the FDA regarding “regulatory flexibility,” uniQure CEO Matt Kapusta stated. “We remain committed to engaging with the FDA to determine a clear, scientifically grounded, and efficient path forward for AMT-130. We are deeply grateful for the resilience and support of the Huntington’s disease community and remain committed to standing with patients and their families.”

 

“Regulatory flexibility” refers to FDA programs that, for example, allow potential accelerated drug approval. uniQure had previously pursued that.

 

Based on its studies so far, which include three years of analysis, uniQure plans to share a four-year analysis, “which we expect to complete in the third quarter of 2026,” Daniel Leonard, the company’s director of global patient advocacy, wrote in a letter e-mailed to the HD community. “We believe the extended follow-up will provide additional insight into both the durability and the magnitude of the effects of AMT-130.”

 

Removing bias from the study

 

Last September, uniQure had reported that AMT-130 demonstrated a 75 percent slowing in the progression of HD over a three-year period. However, in crisis under the Trump administration, the FDA backtracked on its word regarding the firm’s plans to submit its application in early 2026, prompting 48,000-plus people to sign two petitions to the agency asking to keep its promise.

 

On February 24, a top uniQure scientist presented an updated analysis of AMT-130 at the 21st Annual HD Therapeutics Conference.

 

David Margolin, M.D., Ph.D., uniQure’s vice president for clinical development, gave the first scientific presentation at the Therapeutics Conference. He addressed the question, raised by the FDA, regarding the use of data from Enroll-HD, instead of a placebo, in the AMT-130 clinical trial.

 

“We relied on an external comparator cohort, which is selected from participants in the Enroll- HD Natural History Study, which has advantageous characteristics as a comparator,” Dr. Margolin explained. However, whereas the AMT-130 clinical trial takes MRI measurements of the brain, including the crucial striatum, Enroll-HD participants do not.

 

To mitigate the “potential bias” from this difference, uniQure used a statistical technique known as “propensity score matching,” Dr. Margolin stated. As he explained, this involves finding in Enroll-HD individuals similar to those in the uniQure trial and including them as controls.

 

The volume of the striatum (which demonstrates the loss of brain tissue in HD) as a factor in the AMT-130 trial “becomes moot,” Dr. Margolin continued. Other patient characteristics from Enroll-HD “can fully substitute” for that measure, such as neuropsychological testing and measurements of a person’s involuntary movements, he said.

 

“This analysis supports the validity of the three-year efficacy results,” Dr. Margolin concluded. uniQure will publish results of the AMT-130 trial in a scientific journal.

 

Dr. David Margolin (above) displaying a slide demonsrating AMT-130's efficacy in slowing HD progression and (below) enjoying down time at the Therapeutics Conference (photos by Gene Veritas, aka Kenneth P. Serbin)

 

 

‘At the precipice’ of effective treatments

 

Held at the Parker hotel in Palm Springs, CA, the HD Therapeutics Conference concluded on February 26. It is sponsored by CHDI Foundation, Inc., the largest private backer of the quest for HD therapies.

 

Like potential remedies in other HD programs, such as Roche’s tominersen, AMT-130 lowers (reduces) the amount of defective huntingtin protein.

 

“We have multiple shots on goal in the huntington-lowering arena, things like uniQure,” CHDI Chief Scientific Officer Robert Pacifici, Ph.D., told me in an interview on February 27. “While it's true that none of them have made it out the other end with a positive ruling in a pivotal Phase III trial, we're at the precipice of the types of signals that will indicate that there is a clinically meaningful benefit by lowering huntingtin.”

 

Such benefit must be confirmed through a Phase III trial or conditional approval by the FDA.

 

This “exciting” development encourages other companies to invest in late-state trials, Dr. Pacifici added, noting that lowering huntingtin hits at the root causes of HD.

 

 

Dr. Pacifici at the conclusion of the Therapeutics Conference (photo by Gene Veritas)

 

A ‘more conservative’ FDA

 

Cristina Sampaio, M.D., Ph.D., CHDI’s chief medical officer, joined the foundation in 2011 after serving on two entities in the European Medicines Agency (EMA), the FDA’s counterpart in Europe.

 

In a February 25 interview, Dr. Sampaio, who advises companies on their clinical trial programs, observed that companies other than uniQure have faced changes in course with the FDA.

 

Under the Trump administration, she said, the FDA become “more conservative,” especially in the Center for Biologics Evaluation and Research, the unit in charge of evaluating drugs such as AMT-130. News reports have also noted the conservative trend.

 

FDA ‘ping pong’ not healthy

 

At the same time, Dr. Sampaio stated that changes in the FDA’s approval process are “problematic” for uniQure and other firms.

 

“Companies rely on what they discuss with FDA,” Dr. Sampaio said. “This for them is vital. It's how they define their projects, it's how they define how long it will take to the market, it's how they define their budget, how much it will cost.”

 

Changing positions from one day to the next “creates a lot of uncertainty to the market,” she observed. “This ping pong of FDA is not healthy.”

 

As news organizations have reported, changes in leadership at the FDA, firings, and the exit of mid- and high-level managers have created “uncertainty” and “turmoil” at the agency, Dr. Sampaio said.

 

More susceptible to politics

 

Whereas decisions about drugs in Europe tend to be more “democratic,” the FDA is “hierarchical,” making it more susceptible to politics, Dr. Sampaio observed.

 

Even so, she added, outside of normal lobbying, “there was no evidence” until now that political influence was affecting the FDA.

 

“But until these big changes with the Trump administration, the FDA was very respected as an independent body,” Dr. Sampaio asserted.

 

External comparisons are good science

 

Dr. Sampaio noted that the uniQure AMT-130 clinical trial program began in 2019 as an exploratory venture, mainly to test for safety. As time passed and the personnel at the firm changed, the goals for AMT-130 became “more ambitious.”

 

“But this was not pre-planned,” she said. “And this is a weakness. That said, I believe they have been as careful as they can be, given the circumstances, and given the data they have.” The 17 patients about which data have been revealed is also a “small” number, she acknowledged.

 

The decision to use Enroll-HD as a comparison was also first “pushed by the FDA,” Dr. Sampaio noted, albeit under the previous administration.

 

Use of Enroll-HD as an “external comparator” is good science, Dr. Sampaio emphasized, noting that in oncology a similar technique has been used for more than a decade.

 

AMT-130 data moving in the right direction

 

Significantly, all of the AMT-130 data go “in the same direction,” showing improvement in the symptoms, she said. “This is very powerful.”

 

If AMT-130 were granted accelerated approval, it would be conditional, with the drug having to be removed from the market if problems emerged, Dr. Sampaio explained. Accelerated approval would also require ongoing study of the drug for safety and efficacy.

 

Dr. Margolin’s presentation at the therapeutics conference resolved scientific doubt regarding the volume of the striatum, Dr. Sampaio said. “This alleviated the concern that the population in this trial was not representative.”

 

Dr. Sampaio (right) with Dr. Hoa Huu Phuc Nguyen of Ruhr University Bochum, Germany (photo by Gene Veritas)

 

Some think AMT-130 needs more work

 

Blair Leavitt, M.D., a veteran HD researcher and professor of medicine at the University of British Columbia, also weighed in on AMT-130 and the FDA. Dr. Leavitt is the co-founder and co-editor-in-chief of the Journal of Huntington’s Disease.

 

Dr. Leavitt’s lab received funding from uniQure to conduct research.

 

“We tested in our mouse model of Huntington's disease and showed benefits of the uniQure approach in that mouse model,” he explained in a February 26 interview at the Therapeutics Conference. He thinks that the uniQure clinical trial was “designed primarily to show the safety and tolerability of the approach and the agent in individuals with Huntington's disease, and it absolutely did that.”

 

The AMT-130 program has opened “a whole field of gene therapy and gene editing approaches, which is incredibly exciting for our field,” Dr. Leavitt added.

 

However, Dr. Leavitt thinks more work needs to be done on AMT-130. “I would disagree with some people who have suggested [that the work done so far] is sufficient to prove efficacy,” he said, adding that “a proper controlled trial” is ultimately needed. In effect, this was what the FDA has now recommended to uniQure.

 

Regarding the 75 percent slowing of symptoms with AMT-130, Dr. Leavitt observed that “we're talking about a decline in all of the patients, on average, but a less decline. That certainly is positive. That might be the best we can hope for with many of our therapies, but ultimately, it's not a complete reversal of symptoms.”

 

Uncertainty at FDA ‘bad for all’

 

Dr. Leavitt, as a Canadian citizen, declined to comment on the political impact of the Trump administration on the FDA.

 

“But I do believe it is a difficult time for drug development,” he said. “The FDA is really the premier regulator for most of the world, and uncertainty at the FDA leads to uncertainty in drug development, and that's bad for all of us.”

 

Such uncertainty “is the last thing we need at this point, because we are on the precipice of a number of therapies, and I'm sure we're going to have efficacy.”

 

Others firms’ interactions with the FDA

 

Veteran HD researcher Jang-Ho Cha, M.D., Ph.D., the chief science and medical officer of Latus Bio, addressed a question from the audience after his February 24 presentation about the company’s HD program.

 

Ed Wild, M.D., Ph.D.,wanted to know whether Latus had a strategy for engaging with the FDA and its Center for Biologics Evaluation and Research to obtain drug approval. Dr. Wild also asked whether an industry consortium could help “to at least extract some firm guidance?”

 

“Yes, we have a strategy to engage with them,” Dr. Cha responded. Latus hopes to soon submit an Investigational New Drug (IND) application.

 

“At this point, we will wait until we have our IND package to show them everything we’ve got,” Dr. Cha continued. “I’m not sure exactly, if we asked a question from the FDA, we would get an answer that we could rely on exactly at this point. We are watching with a lot of interest other companies who are having interactions with the FDA, because it’s relevant to all of us.”

 

Will the truth conquer all?

 

Dr. Pacifici offered a concluding reflection regarding the FDA.

 

“In terms of the interaction between the companies and the regulators, I guess I'm a bit of an optimist in this regard,” Dr. Pacifici told me. “I truly believe that politics and money and all of the other perverse things that surround us, notwithstanding, that at the end of the day, the truth conquers all.”

 

“I think that these are scientific people,” he said. “I think that they're going to look at the body of data and evidence, and eventually I think they're going to make the very best decision to get things out as quickly and as safely as possible to the patients who so desperately need these treatments.”

 

For additional coverage, visit HDBuzz.

 

Next time: Dr. Pacifici’s overview of the conference and his video interview with me.