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.”

CHDI chief scientist: ‘at the precipice’ of treatments, Huntington’s disease community must ‘persevere’ through the stress

  

As researchers have hypothesized that lowering (reducing) the amount of abnormal huntingtin protein in the brains of people afflicted with Huntington’s disease hits at the root causes of the disease, leading companies have sought to develop drugs in response, like uniQure’s AMT-130 gene therapy.

 

The huntingtin-lowering approach has become central to the search for disease-modifying therapies that slow, halt, or reverse the course of HD, a progressive disorder still without a treatment after the discovery of the huntingtin gene in 1993.

 

"We have multiple shots on goal in the huntington-lowering arena, things like uniQure, like PTC, like Skyhawk,” Robert Pacifici, Ph.D., chief scientific officer for CHDI Foundation, told me in a video 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.”

 

In our interview, Dr. Pacifici gave his customary overview of the CHDI-sponsored 21st HD Therapeutics Conference, held February 23-26 in Palm Springs.

 

Dr. Robert Pacifici (right), interviewed by Gene Veritas, aka Kenneth P. Serbin, February 27, 2026 (screenshot by Gene Veritas)

 

In my introduction, I noted that, because “it had a lot of progress,” scientifically this was the most “exciting” of the conferences that I have attended since my first in 2010.

 

However, I added, “there was stress for us in the HD community about the uniQure gene therapy program” because we were “shocked” at the abrupt backtracking of the U.S. Food and Drug Administration (FDA) on the drug despite its historic efficacy. This prompted two petitions with 48,000-plus signatures. I recalled that “so many of us in the community are still living with the burden of symptoms, including my sister, now disabled.”

 

Dr. Pacifici agreed that, until the arrival of an effective therapy, the HD community will have “some high degree of hope, but also a high degree of stress, because I think the closer we get, the more stressful it is that we're at the precipice of actually realizing this incredible journey.”

 

Dr. Pacific urged that people “continue to persevere.”

 

Watch my full interview with Dr. Pacifici in the video blow. 

 

 

A 75-percent slowing of the disease

 

At the Therapeutics Conference, a uniQure scientist presented an updated analysis of AMT-130 that “supports the validity” of its “efficacy results.”

 

With uniQure’s announcement last September that AMT-130 had demonstrated a 75 percent slowing in the progression of the disease over a three-year period, the HD community was jubilant (click here and here to read more).

 

Reports such as the BBC’s optimistically portrayed AMT-130.

 

HD and uniQure thrust into the national spotlight

 

Observers and news reports have described the Trump administration’s FDA as dysfunctional and more conservative. After the FDA surprisingly reversed field in November regarding AMT-130, the anxiety and raw tensions over the drug underscored the HD community’s yearning for an effective therapy.

 

On March 2 uniQure revealed that the FDA declined to accept its request to keep the agency’s original promise, in which the firm could apply for accelerated approval for AMT-130. Instead, the FDA “strongly recommended” that the company conduct a full-blown, Phase III clinical trial (click here to read more).

 

The controversy over AMT-130 has highlighted the FDA’s rejection of rare-disease drug programs, thrusting HD and uniQure into the national spotlight. Outlets providing coverage have included STAT, The New York Times, Bloomberg, CNN, CNBC, and the The Wall Street Journal.

 

On March 6 Vinay Prasad, M.D., the controversial head of the division within the FDA charged with evaluating gene therapies like AMT-130, resigned for the second time. While no reason was stated, the Times noted, leaders in the biotech and investor communities had long pressed the White House for his ouster.

 

Assisting the 60-plus companies involved

 

Dr. Pacifici told me that he was an “optimist” regarding the “interaction between the companies and the regulators,” who are “scientific people.”

 

“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,” 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.”

 

Because of the progress with huntingtin-lowering drugs like AMT-130, Dr. Pacifici noted that companies continue to allocate the “significant resources” necessary for Phase III trials.

 

The feedback from trials has enabled scientists to “hone the next generation of huntingtin-lowering therapies” and to address related questions, he added.

 

Dr. Pacifici emphasized that CHDI will continue to advise and assist uniQure and all companies seeking to develop HD therapies. More than 60 firms attended the Therapeutics Conference, which had a record attendance of 445.

 

Other key techniques

 

The conference also focused on other potential techniques in the search for therapies, such as the splicing of DNA “either to make less of a toxic protein or make more of a protein that is beneficial,” Dr. Pacifici explained.

 

Several presentations also discussed potential ways of impacting somatic expansion, the tendency of the abnormal huntingtin gene to expand over time to the point where symptoms are triggered.

 

Dr. Pacifici emphasized the need to intervene in the disease process long before symptoms arise.

 

“There's a long phase in Huntington's disease where people have the gene, but overtly, to at least the lay eye, look perfectly normal and healthy,” he explained. However, because they carry the genetic expansion, “deleterious things” can already occur, he added.

 

Another focus included the ongoing study of brain tissue donated by deceased HD-affected individuals, known as post-mortem tissue. This “unbelievably technological marvel of investigations can then look literally cell by cell at those post-mortem brain sections,” Dr. Pacifici explained.

 

Vast data from Enroll-HD

 

Dr. Pacifici and I both wore caps from Enroll-HD, the global registry of more than 22,000 affected individuals and relatives.

 

CHDI runs Enroll-HD, which provides free data to companies seeking to develop therapies.

 

uniQure’s use of Enroll-HD data as an external comparison for AMT-130 clinical trial participants caused the FDA to oppose the company’s request for an accelerated drug approval.

 

Dr. Pacifici called Enroll-HD “one of the most remarkable prospective registry trials that's ever been run.”

 

The Therapeutics Conference included a presentation on Enroll-HD’s whole-genome-sequencing – mapping a person’s entire DNA – of nearly 20,000 of the people in its database. Announced a year ago, this project has already generated 450 terabytes of data.

 

It has revealed some crucial new clues about the genetics of HD onset: new discoveries about potential modifier genes that slow or hasten the start of the disease.

 

Scientists learn much about HD from studying so-called animal models of the disease, but, as Dr. Pacifici reminded me, only humans actually get HD.

 

Enroll-HD safely and non-invasively collects people’s blood, semen, tears, cerebrospinal fluid and DNA and tracks their symptoms, Dr. Padcific said. Enroll-HD thus helps to answer the big question of “how the disease manifests itself in people” over time and how their individual genetics influence the disease.

 

People’s participation in Enroll-HD “makes a huge difference,” Dr. Pacifici observed, adding that the program is positioned to prepare for future research needs as the field evolves.

 

The ‘urgent necessity’ for a therapy

 

On March 5 Amy Gray, the president and CEO of the Huntington's Disease Society of America e-mailed the HD community an update on the controversy involving the FDA, AMT-130, and Enroll-HD.

 

Gray noted the “urgent necessity” of a disease-modifying treatment. She recalled that Congress had directed the FDA to apply “‘regulatory flexibility’ for rare diseases – tailoring approaches when traditional trials are not feasible or would unduly delay access.”

 

Gray added that “innovative trial designs” such as Enroll-HD are appropriate when scientifically justified.

 

“We appreciate uniQure’s stated intent to continue engaging with the FDA,” Gray wrote.

 

Conquering HD

 

Like last year, the 2026 meeting had a session on “new enabling technologies and breakthrough science for neurodegenerative diseases.”

 

“The techniques that people are using would literally have been considered science fiction five years ago,” Dr. Pacifici said.

 

These included a bioengineered “mini-brain,” using actual human cells, that assembled all of the different broad classes of cell types in the brain, Dr. Pacifici explained.

 

The mini-brain was developed and presented at the conference by Alice Stanton, Ph.D., of Massachusetts General Hospital and Harvard Medical School.

 

“We're going to get her to now introduce cells that have the expanded gene and see how that changes so that we could do experiments in a dish instead of in the living organism,” Dr. Pacifici said. "We've got the best, the brightest doing-state-of-the art work."

 

To fill remaining “gaps” and “solve bottlenecks” on the way to therapies, yet more discoveries will be needed, he added.

 

With CHDI’s multi-million-dollar resources, the involvement of big companies, and the participation of HD families, “we’re going to conquer this thing,” Dr. Pacifici predicted.

 

 

Dr. Alice Stanton presenting her talk on her human "mini-brain" invention (above) and taking questions from the audience (below) (photos by Gene Veritas)

 

 

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.