On Thursday, FDA released new policies on opioids in an unsuccessful effort to wipe away opposition in the U.S. Senate to Robert Califf's confirmation as FDA commissioner. Califf, who is deputy commissioner for medical products and tobacco, is the only FDA official quoted in a press release announcing the agency's Opioid Action Plan. Sens. Edward Markey (D-Mass.) and Joe Manchin (D-W.Va.) said the new policies are welcome, but fall short of meeting their conditions for dropping holds they have placed on a Senate vote to confirm Califf (see BioCentury, Feb. 1).
According to a fact sheet, FDA will "fundamentally re-examine the risk-benefit paradigm for opioids and ensure that the agency considers the wider public health effects." It is seeking advice from the Science Board in March, and has already asked the National Academy of Medicine to provide recommendations on "how to take into account our evolving understanding of the risks of opioids, not only to the patient but also the risks of misuse by other persons who obtain them."
The agency committed to submit applications for any opioids that do not have abuse-deterrent properties to an advisory committee, and to solicit recommendations from its Pediatric Advisory Committee "regarding a framework for pediatric opioid labeling before any new labeling is approved." FDA said it will consult an advisory committee on abuse-deterrent formulation opioids when the products raise novel issues.
The agency said it will issue draft guidance with its recommendations on approval standards for generic abuse-deterrent formulations. It also is developing new warnings and safety information for immediate-release opioids consistent with those it imposed on extended-release opioids in 2013 and is strengthening requirements for postmarket studies of long-term use of extended-release/long-acting opioids.
Additional elements of FDA's opioid action plan include overhauling REMS requirements and reviewing policies to make naloxone, an antidote for opioid overdoses, more widely available. This could include making naloxone available without a prescription.
In a statement, Markey said he will continue to try to block Califf's confirmation because the new policies comply with some, but not all of his requests. He said the action plan includes "important steps to address the opioid crisis, but they fall short of what is needed." Markey said FDA "must change its decision not to seek expert advice about the risks of addiction before it approves abuse-deterrent opioids." He added: "Until the FDA commits to convene advisory committees of outside experts for all its opioid approval decisions, I will continue my hold on Dr. Califf's nomination."
Manchin went further, saying in a statement that he will oppose Califf's confirmation unless FDA agrees to not only hold advisory committee meetings for all opioid reviews, but also to adhere to any recommendations made by the committees. Manchin made it clear that his goal is to prevent approvals of new opioids. "I will continue to strongly pressure the FDA to strengthen its oversight of opioid medications and will continue to push for the strongest possible framework for drug approval to ensure that the agency doesn't simply continue to approve ever stronger and more deadly opioid medications under this new process."
Under Senate procedures, Majority Leader Sen. Mitch McConnell (R-Ky.) could schedule a vote to override the holds. Sixty votes would be needed to override the holds, and 50 votes would be needed to confirm Califf as FDA commissioner.
Stanford University (Stanford, Calif.) hired Marc Tessier-Lavigne as president, effective Sept. 1. He will succeed John Hennessy, who last year announced plans to step down as president.
Tessier-Lavigne has been president of The Rockefeller University since 2011. He is a neuroscientist who was a professor of biological sciences at Stanford from 2001-05, though he was on leave for the latter years while he worked at the Genentech Inc. unit of Roche (SIX:ROG; OTCQX:RHHBY). He held several positions at Genentech, including EVP for research and CSO.
Tessier-Lavigne also helped co-found and is chairman of neurodegenerative disease company Denali Therapeutics Inc. (South San Francisco, Calif.).
Rockefeller said it is forming a search committee for a new president.
FDA's Psychopharmacologic Drugs Advisory Committee voted 8-2 on Wednesday that H. Lundbeck A/S (CSE:LUN) and partner Takeda Pharmaceutical Co. Ltd. (Tokyo:4502) provided substantial evidence to support an efficacy claim for Brintellix vortioxetine to treat cognitive dysfunction in adults with major depressive disorder (MDD). The companies market Brintellix in the U.S. to treat MDD in adults.
The committee was discussing an sNDA from Takeda seeking to add the claim to Brintellix's label. The drug is a serotonin modulator and stimulator. The PDUFA date for the sNDA is March 28, 2016.
In an update provided to Parent Project Muscular Dystrophy, Eli Lilly and Co. (NYSE:LLY) said a Phase III trial of tadalafil (LY450109) to treat Duchenne muscular dystrophy (DMD) "provided no evidence that once-daily tadalafil treatment has a meaningful effect to slow disease progression compared with placebo." The double-blind trial enrolled about 330 DMD patients.
Lilly told the DMD patient group that it "did not see any evidence of efficacy for tadalafil to slow the decline" on the primary endpoint of six-minute walking distance (6MWD) through 48 weeks. Additionally, Lilly said there was no evidence of efficacy on secondary endpoints assessing motor function, including the North Star Ambulatory Assessment and timed function tests.
According to a presentation by LLY, the pharma was evaluating whether tadalafil could correct dysfunctional muscle blood flow by amplifying the nitric oxide (NO)/cGMP pathway.
Tadalafil, which is marketed as Cialis and Adcirca, is approved to treat erectile dysfunction, pulmonary arterial hypertension (PAH) and signs and symptoms of benign prostatic hyperplasia (BPH).
Lilly said it will submit the Phase III DMD data for presentation at scientific and patient advocacy meetings, and will submit the data for publication. The pharma also halted an open-label extension of the Phase III trial.
Imperial Innovations Group plc (LSE:IVO) plans to raise L100 million ($142.9 million) through a placing of 23.5 million shares at 425p to existing and new institutional investors. The price is a 6% premium to the firm's close of 400p on Wednesday, before the offering was proposed. The placing is subject to shareholder approval by May 31.
Imperial Innovations acts as the technology transfer office for Imperial College London and also invests in technologies from the University of Oxford, the University of Cambridge and University College London.
The company said it plans to invest the new funds in its existing and future portfolio companies, its commercialization and investment activities in technologies, in investment opportunities that arise from the firm's involvement in the UCL Technology Fund, and to expand its licensing portfolio to grow its license and royalty income.
UCL launched the UCL Technology Fund last month with L50 million ($71.5 million). Imperial Innovations invested L24.8 million ($35.4 million) (see BioCentury Extra, Jan. 19).
Imperial Innovations was up 20.5p to 420.5p on Thursday.
Prodrug developer KemPharm Inc. (NASDAQ:KMPH) raised $75 million through the sale of senior unsecured notes in a deal led by Deerfield Management. Book-running managers were Cowen and RBC Capital Markets, and co-managers were Canaccord and Oppenheimer.
The 5.5% notes are due in 2021. In December, KemPharm proposed to raise up to $62.7 million in a follow-on through the sale of 3 million shares, but withdrew the offering on Tuesday.
On Thursday, KemPharm slipped $0.02 to $14.80.
More than 230 leaders of the biopharma industry have signed an open letter expressing outrage and calling for an end to the use of "scantily clad" female models and dancers at professional networking events.
The letter was circulated by Kate Bingham and Karen Bernstein in response to the LifeSci Advisors After Party that occurred during the week of the JPMorgan Healthcare Conference in San Francisco, "where young, female models were brought in to escort the guests on buses to the Exploratorium and to mingle with the crowd," according to the letter.
JPMorgan was not affiliated with the LifeSci Advisors event.
The letter also notes that "the official video of the 27th Annual ROTH Conference proudly flaunts its use of scantily clad female dancers."
Bingham is managing partner at SV Life Sciences Advisers. Bernstein is co-founder and chairman of BioCentury Publications, Inc.
In a statement emailed to BioCentury, LifeSci Advisors co-founders Michael Rice and Andrew McDonald said, "LifeSci Advisors made a serious mistake when we invited female promotional models to our after party following the JP Morgan Healthcare Conference in San Francisco last month.
"We want to apologize to our female and male colleagues throughout the biotech and bioscience industries. In addition, the open letter that Kate Bingham and Karen Bernstein wrote this week has had a positive impact on us and on our industry.
"As a firm, we now have an opportunity to make a difference and help tackle some of the core issues facing women in our field. Some of those issues include the lack of women in management and leadership positions, the lack of mentors and professional development networks for women that are necessary to cultivate future leaders in our industry and the underrepresentation of girls in STEM programs.
"In the coming days, LifeSci Advisors will be launching a series of concrete initiatives to address these systemic issues. We plan to invest our time, energy, resources and money to help resolve these problems and hopefully play a meaningful role in improving our industry's track record on diversity."
Bingham said, "This is absolutely the right thing for LifeSci Advisors to have done, and we look forward to working with them on their initiatives to support diversity and inclusion. But no less importantly, we encourage others engaging in these unsavory practices to follow in their footsteps."
Bernstein added, "If we have truly put an end to these kinds of activities in our industry, it will be one small step for humanity and I will consider it well worth the efforts of all the people who supported this -- including all the people whose signatures we didn't have the time to add."
John Chambers, vice chairman and head of healthcare investment banking at Roth Capital Partners, told BioCentury, "It's unfortunate to have our name referenced in this context by people who have not attended our event. We have more families and spouses attend than any other investor conference I've been involved in. I haven't had a single complaint from anyone who's attended. I'm proud of the event for the visibility it provides to emerging growth companies to gain access to investors in a professional setting."
The Roth event included performances by the rock groups Poison and Fall Out Boy.
The open letter was reported by FierceBiotech on Feb. 2, and elicited a strong response on Twitter. BioCentury did not ask its employees to sign the letter, but co-founder, President and CEO David Flores, BioCentury Editor Susan Schaeffer and BioCentury Innovations Editor C. Simone Fishburn have signed.
Janet Woodcock, director of FDA's Center for Drug Evaluation and Research, told members of the U.S. House Energy and Commerce Committee's health subcommittee she is concerned about the agency's ability to manage what she expects will be an increasing number of biosimilars applications. "What I'm concerned about is that this program is going to explode," said Woodcock. "I'm concerned that we will not have the staff because we're always waiting to catch up."
Woodcock's comments came in response to a question from Rep. Frank Pallone (D-N.J.) about whether and how lack of funding is affecting FDA's ability to implement the Biologics Price Competition and Innovation Act (BPCIA). Pallone is the ranking Democrat on the E&C committee.
The health subcommittee was holding a hearing on Thursday to discuss the implementation of BPCIA, which was enacted in 2010.
In her written testimony, Woodcock said that as of Jan. 21, 59 proposed biosimilar products for 18 different reference products were enrolled in the agency's Biosimilar Product Development (BPD) Program. The number of meeting requests increased to 58 in FY15 from 32 in FY13, and the number of scheduled meetings increased to 50 in FY15 from 30 in FY13.
Along with the increase in meetings, Woodcock said FDA's FTE expenditure in the first two quarters of FY15 was equivalent to the agency's total FTE expenditure in FY14. "The increase in FTE expenditure is a direct reflection of the change and increase in workload in FY2015," wrote Woodcock.
"It sounds to me like you need some kind of appropriation. Just having the industry pay a fee isn't good enough," Pallone told Woodcock. At the hearing, she said FDA collected $6 million in user fees in FY13, $13 million in FY14, and $23.8 million in FY15.
In response to a separate question, Woodcock said FDA would review an application for interchangeability before it had issued guidance on the topic. Last month, CDER said it plans to release long-awaited guidance on interchangeability and labeling of biosimilars this year.
Sean Cavanaugh, who is deputy administrator and director of CMS's Center of Medicare, also testified at Thursday's hearing. Members of the health subcommittee questioned Cavanaugh at length about the agency's coding and billing policy for biosimilars.
Starting Jan. 1, a final rule from CMS took effect that sets a single reimbursement price for all biosimilars of a given reference product covered under Medicare Part B (see BioCentury Extra, Oct. 30, 2015).
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