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BioCentury Extra
As published Friday, November 21, 2014 6:24 PM PST

  • Rep. Upton updates 21st Century Cures timeline

    Rep. Fred Upton (R-Mich.) said he plans to release a discussion draft in mid-January 2015 of legislation based on the 21st Century Cures initiative he and Rep. Diana DeGette (D-Colo.) launched in May.

    Speaking at a conference sponsored by Friends of Cancer Research and the Brookings Institution, Upton said his goal is to enact a law that will speed the development of drugs and devices to treat unmet medical needs. Upton didn't reveal details of the legislation, but said it will include ways to increase funding for NIH and FDA and will create more opportunities to support young scientists.

    The House Energy and Commerce Committee chairman said he will release the draft in the second or third week of January, then introduce the legislation "a couple of weeks" later. He said he plans to "move it through the committee before the end of March and have it on the [House] floor before Memorial Day."

    The shift to Republican control of the Senate increases the likelihood that the 21st Century Cures legislation will be enacted because it would give Senate Republican leadership a bipartisan accomplishment (see BioCentury, Nov. 10, 2014).

    Upton called on patient advocates to rally support for the legislation, saying that a large bipartisan majority in the House would enhance the bill's chances in the Senate. Upton said he met Thursday with Sen. Orrin Hatch (R-Utah), incoming chair of the Senate Finance Committee, and has scheduled additional meetings with senators during the first week of December to line up support.

    Upton and DeGette discussed the launch of 21st Century Cures on BioCentury This Week television in June (see BioCentury This Week, June 1).

  • CHMP gives nod to AbbVie's HCV therapies

    EMA's CHMP recommended approval of Exviera dasabuvir and Viekirax, which is a two direct-acting-antiviral (2-DAA) combination of ombitasvir and paritaprevir (ABT-450) plus the booster Norvir ritonavir from AbbVie Inc. (NYSE:ABBV) to treat chronic HCV infection.

    CHMP recommended the 3-DAA combination of Viekirax plus Exviera, with or without ribavirin, to treat HCV genotype 1, and recommended Viekirax plus ribavarin to treat HCV genotype 4. EMA is evaluating MAAs for both compounds under accelerated assessment and AbbVie expects a final decision by 1Q15. CHMP said marketing authorization for both products would include a pharmacovigilance plan.

    The combination regimen of ombitasvir, paritaprevir and Norvir has breakthrough therapy designation from FDA and is under Priority Review for HCV genotype 1. AbbVie spokesperson Jaquelin Finley told BioCentury the company expects FDA approval by year end, and said the Exviera and Viekirax names will be used only in the EU. AbbVie submitted the NDA in April (see BioCentury Extra, April 22).

    Paritaprevir, an HCV NS3/4A protease inhibitor, is the lead HCV protease inhibitor discovered under a 2006 deal between Enanta Pharmaceuticals Inc. (NASDAQ:ENTA) and AbbVie predecessor Abbott Laboratories. AbbVie is responsible for development and commercialization of paritaprevir.

    Ombitasvir is an HCV NS5A protein inhibitor and dasabuvir is a non-nucleoside HCV NS5B polymerase inhibitor. AbbVie markets Norvir, an HIV protease inhibitor.

  • CHMP backs psoriasis treatments from Novartis, Celgene

    EMA's CHMP recommended marketing authorization for Cosentyx secukinumab from Novartis AG (NYSE:NVS; SIX:NOVN) as a first-line treatment for adults with moderate to severe plaque psoriasis and Otezla apremilast from Celgene Corp. (NASDAQ:CELG) to treat active psoriatic arthritis and moderate to severe chronic plaque psoriasis in adults. CHMP said marketing authorization for both products would include a pharmacovigilance plan.

    Last month, FDA's Dermatologic and Ophthalmic Drugs Advisory Committee voted unanimously to recommend approval of Cosentyx for moderate to severe plaque psoriasis. Novartis submitted a BLA for the human IgG1 mAb targeting IL-17 in October 2013; the PDUFA date is not disclosed. The compound is the first therapy targeting IL-17 recommended for approval in Europe for this indication.

    FDA approved an NDA for Otezla in March to treat adults with active psoriatic arthritis and an sNDA in September for moderate to severe plaque psoriasis. Otezla is an oral phosphodiesterase-4 (PDE-4) inhibitor.

  • CHMP supports Boehringer's nintedanib for IPF

    EMA's CHMP recommended marketing authorization of Ofev nintedanib from Boehringer Ingelheim GmbH (Ingelheim, Germany) for the treatment of idiopathic pulmonary fibrosis (IPF). The compound is under accelerated assessment in the EU.

    In September, CHMP recommended approval of nintedanib plus docetaxel to treat locally advanced, metastatic or locally recurrent non-small cell lung cancer (NSCLC) with adenocarcinoma histology after failure of first-line chemotherapy (see BioCentury Extra, Sept. 26).

    Last month, the FDA approved Ofev to treat IPF. It has Orphan Drug and breakthrough therapy status in the U.S. and Orphan Drug designation in Japan for IPF (see BioCentury Extra, Oct. 15).

    CHMP said marketing authorization would include a pharmacovigilance plan in IPF. It made the same recommendation in NSCLC.

    The drug is a small molecule inhibitor of multiple pro-angiogenic kinases, including VEGF, platelet derived growth factor receptor (PDGFR) and fibroblast growth factor (FGF) receptor (FGFR).

  • Genzyme's Cerdelga gets CHMP nod for Gaucher's

    EMA's CHMP recommended marketing authorization of Cerdelga eliglustat tartrate from the Genzyme Corp. unit of Sanofi (Euronext:SAN; NYSE:SNY) to treat Type I Gaucher's disease.

    Cerdelga is an oral ceramide analog that inhibits glucosylceramide synthase (GCS).

    CHMP said the marketing authorization would include a pharmacovigilance plan.

    Cerdelga is under standard review in the EU. In August, FDA approved Cerdelga to treat Type I Gaucher's disease after Priority Review. It has Orphan drug designation in Japan (see BioCentury Extra, Aug. 19).

    Genzyme also markets Cerezyme imiglucerase, a recombinant glucocerebrosidase, for the same indication.

  • Boehringer, WellPoint studying outcomes in AF

    Boehringer Ingelheim GmbH (Ingelheim, Germany) and the HealthCore Inc. unit of WellPoint Inc. (NYSE:WLP) have identified non-valvular atrial fibrillation (NVAF) as the first project under their July deal to collaborate on the collection of real world health economic and outcomes data.

    HealthCore, which is Wellpoint's outcomes research arm, and BI said they chose to study non-valvular AF because new anticoagulant therapies have begun to replace warfarin.The partners plan to study disease prevalence, treatment complications such as hospital readmissions and the use of evidence-based guidelines.

    The pharma markets its Pradaxa dabigatran etexilate in the U.S. and EU to reduce the risk of stroke and blood clots in patients with NVAF and other indications.

    Boehringer spokesperson Mary Lewis told BioCentury the pharma is considering additional therapeutic areas to study under the five-year WellPoint deal, including cancer, respiratory and metabolic conditions.

    HealthCore is also partnered with AstraZeneca plc (LSE:AZN; NYSE:AZN) to conduct health outcomes research under a 2011 deal (see BioCentury, Aug. 1, 2011).

  • G-BA: No additional benefit for Tecfidera in MS

    Germany's Federal Joint Committee (G-BA) has concurred with the Institute for Quality and Efficiency in Healthcare (IQWiG) that Tecfidera dimethyl fumarate from Biogen Idec Inc. (NASDAQ:BIIB) offers no additional benefit over interferon beta-1a to treat relapsing-remitting multiple sclerosis (RRMS).

    G-BA's final assessment was published Thursday, and mirrored the finding IQWiG reached in August (see BioCentury Extra, Aug. 4).

    Biogen spokesperson Catherine Falcetti said G-BA's decision "forms the basis for negotiating a final price" of Tecfidera with Germany's Federal Association of Statutory Health Insurance Funds.

    Tecfidera is an oral dimethyl fumarate that activates the NF-E2-related factor 2 (Nrf2) pathway.

    Separately, EMA's CHMP asked Biogen Idec to send letters to healthcare professionals alerting them to a potential risk of progressive multifocal leukoencephalopathy (PML) associated with use of Tecfidera. Last month, the company announced that a patient who had been treated with Tecfidera for over four years developed PML and ultimately died of pneumonia (see BioCentury Extra, Oct. 22).

    Biogen finished up $4.45 to $303.55 on Friday.

  • Lexicon raises $130M in follow-on and notes

    Lexicon Pharmaceuticals Inc. (NASDAQ:LXRX) raised $80 million through the sale of senior convertible notes and $50 million in a follow-on through the sale of 49.8 million shares at $1. JP Morgan; Goldman Sachs; Needham; and Stifel, Nicolaus are underwriters.

    The notes bear 5.25% interest, mature in 2021 and initially convert at $1.21.

    Lexicon expects top-line data in 3Q15 from a Phase III study of lead compound telotristat etiprate to treat carcinoid syndrome, which is associated with carcinoid tumors. The tryptophan hydroxylase inhibitor has Orphan Drug and Fast Track designations in the U.S. for this indication and Orphan Drug designation in the EU for carcinoid tumors.

    Lexicon fell $0.06 to $0.95 on Friday.

  • Banker tracks

    Dresner Partners hired Robert Friedman as managing director, healthcare. Friedman was most recently managing director at Northeast Securities, and previously co-head of healthcare investment banking for Burnham Securities.

  • Woodcock: Breakthrough designation mostly about efficacy

    FDA awards or denies breakthrough designation requests primarily based on convincing clinical evidence of substantial efficacy or lack thereof, Janet Woodcock said Friday at a conference organized by Friends of Cancer Research and the Brookings Institution. Woodcock is director of the agency's Center for Drug Evaluation and Research.

    In a review of the breakthrough program's first two years, FDA found that a 50% reduction of a key risk -- for example, disease progression -- would suffice to merit a breakthrough designation, she said.

    "When the endpoint is survival, lesser improvements are still impressive," she added. "In general, improvements of 10% over a comparator do not seem to be breakthrough territory."

    Woodcock reported that CDER cited lack of efficacy as the reason for 66% of breakthrough denials, and lack of safety for 16%. Other reasons included a lack of clinical data. CDER has granted 62 of 163 breakthrough requests; two more were withdrawn before CDER acted on them. The Center for Biologics Evaluation and Review has granted seven of 37 requests.

    Targeted therapies are more likely to obtain breakthrough designations. "Therapies with a genetic component in their indication were more likely than those without one to be granted breakthrough status, but Orphan and/or rare status did not make a difference," Woodcock said.

    Woodcock said the large number of designations reflects broad, continuing advances in science and drug development. "There is no letup in applications, so I expect a robust program going forward," she said.

    While Woodcock said it would be premature to evaluate the overall impact of the breakthrough program on drug development, she said a "number of breakthrough-designated drugs have undoubtedly reached patients sooner" because of the process.

    The breakthrough program is resource-intensive, Woodcock said, but its strain on CDER resources is difficult to ascertain because the center is broadly understaffed. CDER has 650 unfilled vacancies, she reported.

    Woodcock discussed the breakthrough program in an interview on BioCentury This Week television in June (see BioCentury This Week, June 8).

  • CMS issues final guidance on CED program

    The Centers for Medicare & Medicaid Services (CMS) issued final guidance on its coverage with evidence development (CED) program that clarifies the program's stopping rules.

    CMS uses CED to extend coverage to drugs and devices conditionally, contingent on some form of clinical data collection, typically via clinical trials or patient registries.

    The final guidance says CMS can remove the requirement for study participation through a reconsideration process, generally following publication of relevant study data. It suggests that sponsors supply interim analyses to CMS, and that CMS is empowered to revise and expedite coverage decisions based on interim analyses.

    Industry stakeholders were concerned that the 2012 draft guidance lacked a transitional pathway to full coverage for CED medicines and devices, thereby limiting their use indefinitely (see BioCentury, April 9, 2012).

    CMS did not return calls asking about the timelines associated with standard and expedited decisions.

  • FDA to hold workshop on LDT guidance

    FDA will hold a public workshop on Jan. 8 and 9 in Bethesda, Md., to discuss its proposal for a risk-based framework to address regulatory oversight of laboratory developed tests (LDTs). The agency plans to discuss with stakeholders how to balance patient safety and access to LDTs.

    FDA released two draft guidance documents in September outlining its plans for LDT regulation (see BioCentury Extra, Sept. 30).

  • FDA workshop on therapies for rare patient subsets

    FDA will hold a public workshop on Dec. 12 to discuss approaches to evaluate precision therapies targeting subsets of patients with rare molecular characteristics. The Center for Translational and Regulatory Sciences at the University of Virginia will co-sponsor the workshop, entitled "Developing and Using Precision Therapies in the 'Omics' Era: Generating and Interpreting Evidence for Rare Subsets."

    The agency and stakeholders will discuss how to obtain evidence that can be used to evaluate therapies when rare patient subsets are identified through in vitro diagnostic testing, in cases when specific, controlled trials are not feasible.

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