FDA approved Viekira Pak from AbbVie Inc. (NYSE:ABBV) with or without ribavirin to treat HCV genotype 1 infection in patients with and without compensated cirrhosis. AbbVie set the WAC for the three direct-acting-antivral (3-DAA) treatment regimen at $83,319 for a 12-week course.
Viekira Pak includes two tablets: a fixed-dose combination of 25 mg of ombitasvir (ABT-267), 150 mg of paritaprevir (ABT-450), and 100 mg of the booster Norvir ritonavir dosed once daily; and 250 mg of dasabuvir (ABT-333) dosed twice daily. The regimen also includes ribavirin for patients with HCV genotype 1a, and all genotype 1 patients who have cirrhosis or have received a liver transplant.
Viekira Pak's label recommends a 12-week course for HCV genotype 1a patients without cirrhosis and HCV genotype 1b patients with or without cirrhosis. HCV genotype 1a patients with cirrhosis are dosed for 24 weeks.
In October, Gilead Sciences Inc. (NASDAQ:GILD) launched its 12-week course of Harvoni ledipasvir/sofosbuvir at a WAC of $94,500.
Harvoni is a single pill dosed once daily, consisting of 90 mg of the HCV NS5A protein inhibitor ledipasvir, plus 400 mg of Gilead's nucleotide analog HCV NS5B polymerase inhibitor Sovaldi sofosbuvir. Harvoni is also approved as an 8-week regimen, which Gilead said could be used in about half of HCV patients at a WAC of $63,000.
Gilead has faced criticism for the prices of Harvoni and Sovaldi, which is approved in combination with other therapies to treat HCV genotypes 1, 2, 3 and 4. A 12-week course of Sovaldi has a WAC of $84,000 (see BioCentury, Oct. 6).
The paritaprevir component of Viekira Pak was discovered under a 2006 deal with Enanta Pharmaceuticals Inc. (NASDAQ:ENTA), which will receive a $75 million milestone as a result of the approval.
EMA's CHMP recommended approval of AbbVie's combination last month for HCV genotypes 1 and 4 infection. EMA is evaluating an MAA for the regimen under accelerated assessment and AbbVie expects a final decision by 1Q15 (see BioCentury Extra, Nov. 21).
Ombitasvir is an HCV NS5A protein inhibitor and dasabuvir is a non-nucleoside HCV NS5B polymerase inhibitor. AbbVie markets Norvir, an HIV protease inhibitor.
AbbVie said it will offer a patient assistance program to provide Viekira Pak at no cost to patients with financial difficulties and a co-pay assistance program for commercially-insured patients.
AbbVie lost $0.21 to $67.71 on Friday. Enanta rose $1.74 to $46.62.
FDA approved Zerbaxa ceftolozane/tazobactam from Cubist Pharmaceuticals Inc. (NASDAQ:CBST) to treat adults with complicated intra-abdominal infections (cIAI) and complicated urinary tract infections (cUTI).
Zerbaxa was under Priority Review after receiving Qualified Infectious Disease Product designation under the Generating Antibiotic Incentives Now (GAIN) Act to treat Gram-negative bacteria. QIDP qualifies the drug for an additional five years of marketing exclusivity.
Zerbaxa -- a combination of the cephalosporin antibiotic ceftolozane (CXA-101) and the beta lactamase inhibitor tazobactam -- also is under EMA review.
The drug's label contains a warning that it is less effective in patients with renal impairment.
In 2015, Cubist expects Phase III data for Zerbaxa to treat hospital-acquired bacterial pneumonia (HABP) and ventilator-associated bacterial pneumonia (VABP).
The news came after market close. The biotech's shares finished up $0.90 to $98.67 on Friday, still below the $102 per share cash portion of its deal to be acquired by Merck & Co. Inc. (NYSE:MRK) for $8.4 billion in cash plus assumption of debt for a total deal value of $9.5 billion (see BioCentury Extra, Dec. 8).
FDA granted accelerated approval to Lynparza olaparib from AstraZeneca plc (LSE:AZN; NYSE:AZN) as monotherapy in patients with deleterious or suspected deleterious germline BRCA mutated advanced ovarian cancer, as detected by an FDA-approved test, who have been treated with three or more prior lines of chemotherapy.
AstraZeneca spokesperson Michelle Meixell told BioCentury the company expects to launch the drug by year end. Meixell would not disclose its price.
FDA approved the poly(ADP-ribose) polymerase (PARP) inhibitor in conjunction with companion diagnostic BRACAnalysis CDx from Myriad Genetics Inc. (NASDAQ:MYGN).
AstraZeneca said full U.S. approval is contingent on a review of survival data from two ongoing confirmatory Phase III SOLO trials. Data from the SOLO2 study are expected next year, and from SOLO3 in 2019.
AstraZeneca originally sought FDA approval of Lynparza as monotherapy for the maintenance treatment of women with platinum-sensitive relapsed ovarian, fallopian tube or primary peritoneal cancer with gBRCA mutation, as detected by an FDA-approved test, who have responded to platinum-based chemotherapy. In June, FDA's Oncologic Drugs Advisory Committee (ODAC) voted 11-2 that the company's safety and efficacy data did not support accelerated approval for this indication (see BioCentury Extra, June 25).
Subsequently, AstraZeneca amended its NDA and submitted additional data from a Phase II study supporting Lynparza for its FDA-approved indication.
On Thursday, the European Commission approved Lynparza as maintenance treatment for platinum-sensitive relapsed BRCA-mutated ovarian cancer patients who have responded to chemotherapy.
Myriad gained $0.85 to $36.42 on Friday.
Adocia S.A. (Euronext:ADOC) rose EUR 10.16 (37%) to EUR 37.40 after granting Eli Lilly and Co. (NYSE:LLY) exclusive, worldwide rights to BioChaperone Lispro to treat Type I and Type II diabetes.
Adocia will receive $50 million up front and is eligible for up to $280 million in development and regulatory milestones and up to $240 million in sales milestones, plus tiered royalties. Lilly will reimburse Adocia for certain R&D expenses under the deal, and pay for development and commercialization.
BioChaperone Lispro is an ultra-fast acting formulation of insulin lispro using Adocia's BioChaperone polymer technology. Adocia said the technology increases a protein's solubility, protects against enzyme degradation and extends the protein's action time.
A formulation corresponding to a 100 IU/ml dose of Lilly's Humalog insulin lispro, BioChaperone Lispro U100, is in clinical testing. Lilly defines the candidate's stage of development as Phase Ib, while Adocia describes it as Phase I/II. Adocia CEO Gerard Soula said the company has preclinical U200 and U300 formulations as well.
Last year, Adocia and Lilly mutually terminated a 2011 deal to develop and commercialize a version of Humalog using BioChaperone. Adocia retained IP covered under the deal, and continued work on the project. Soula said the companies had a strategic disagreement and that Lilly de-prioritized the project. Lilly spokesperson Greg Kueterman said Lilly's termination of the deal was a "difficult financial decision."
In September, Adocia said that in a Phase IIa trial, 0.2 U/kg BioChaperone Lispro led to a significantly faster rate of insulin lispro absorption as measured by AUC (p<0.001) and a significantly lower median time to peak insulin concentration (Tmax, p=0.001) compared to Humalog in Type I diabetes patients.
Kueterman declined to disclose a development timeline for the compound.
Lilly paid Adocia $10 million up front in the 2011 deal.
Ono Pharmaceutical Co. Ltd. (Tokyo:4528) granted Gilead Sciences Inc. (NASDAQ:GILD) exclusive rights to develop and commercialize ONO-4059 outside of Japan, South Korea, Taiwan, China and countries in the Association of Southeast Asian Nations (ASEAN), where Ono retains development and commercialization rights. Ono will receive an undisclosed upfront payment and is eligible for milestones.
Gilead plans to develop the Bruton's tyrosine kinase (Btk) inhibitor to treat B cell malignancies and other undisclosed conditions. Ono said the molecule has shown activity in Phase I testing in chronic lymphocytic leukemia (CLL) and non-Hodgkin's lymphoma (NHL).
Gilead rose $2.91 to $108.45 on Friday. Ono gained Y220 to Y10,390.
EMA's CHMP recommended conditional marketing authorization for Holoclar from Chiesi Farmaceutici S.p.A. (Parma, Italy) to treat adults with moderate to severe limbal stem cell deficiency (LSCD) due to physical or chemical burns to the eyes.
Holoclar is the first therapy CHMP has recommended for LSCD, and the first advanced therapy medicinal product (ATMP) containing stem cells it has recommended.
Holoclar consists of ex vivo expanded autologous human corneal epithelial cells containing stem cells. The laboratory-grown therapy is made from a biopsy from an undamaged area of the cornea, then transplanted into one or both of a patient's affected eyes.
CHMP based its recommendation on a draft opinion of the Committee for Advanced Therapies (CAT), EMA's expert committee for ATMPs. CAT and CHMP said existing data are not comprehensive, and recommended an additional study on Holoclar's use.
Orexigen Therapeutics Inc. (NASDAQ: OREX) stands to be first to market with a new obesity drug in Europe as EMA's CHMP adopted a positive opinion for Mysimba naltrexone/bupropion as an adjunct to reduced-calorie diet and increased physical activity for the management of weight in adults who are obese or are overweight with weight-related co-morbidities.
CHMP recommended treatment be stopped after 16 weeks if the patient has not lost at least 5% of initial body weight.
The EC is expected to issue a final marketing authorization decision in early 2015.
The drug is marketed as Contrave in the U.S. In September, it became the third new drug to win FDA approval for obesity since 2012, following Belviq lorcaserin from Arena Pharmaceuticals Inc. (NASDAQ:ARNA) and Eisai Ltd. (Tokyo:4523) and Qsymia phentermine/topiramate from Vivus Inc. (NASDAQ:VVUS) in July 2012.
But in Europe, Arena withdrew its MAA for Belviq in 2013 after CHMP asked the biotech to justify the compound's benefit-risk balance given preclinical and clinical issues including tumors in rats, valvulopathy and undisclosed psychiatric events (see BioCentury Extra, May 2, 2013).
Also in 2013, CHMP issued a negative opinion for Qsymia, citing potential cardiovascular and CNS effects associated with long-term use and asking for a preapproval cardiovascular outcomes trial (see BioCentury Extra, Feb. 21, 2013).
Orexigen is conducting a post-approval CV outcomes trial to meet FDA's approval requirements, which CHMP noted in its announcement.
Orexigen partner Takeda Pharmaceuticals Co. Ltd. (Tokyo:4502) is marketing the drug in the U.S. The biotech has not yet partnered the drug outside North America.
Orexigen shares added $0.04 to $6.33 on Friday.
EMA's CHMP recommended marketing authorization of safinamide from Newron Pharmaceuticals S.p.A. (SIX:NWRN) and partner Zambon Co. S.p.A. (Bresso, Italy) as add-on therapy to a stable dose of a dopamine agonist for patients with early PD, and as add-on therapy to levodopa alone or in combination with other treatments in patients with mid- to late-stage PD.
CHMP said the marketing authorization will include a pharmacovigilance plan.
Safinamide is an alpha-aminoamide derivative that acts as a reversible monoamine oxidase B (MAO-B) and dopamine reuptake inhibitor while reducing glutamatergic activity.
Newron submitted an NDA to FDA in June. Zambon has exclusive rights from Newron to develop and commercialize safinamide worldwide, excluding Asian territories where Meiji Seika Pharma Co. Ltd. (Tokyo, Japan) has rights.
Newron closed up CHF3.15 to CHF26.45 on Friday.
Three days after FDA approved the Intercept Blood System for plasma, Cerus Corp. (NASDAQ:CERS) announced the agency approved the company's Intercept system for platelets. Cerus shares rose $0.25 to $6.41 on Friday; they have gained 34% since Tuesday's close, before the plasma system approval was announced.
Cerus already markets the system for plasma and for platelets in Europe, the Commonwealth of Independent States and the Middle East.
The platelet system uses light-activated molecules to crosslink DNA and RNA to prevent pathogen replication. It is currently available under an expanded access investigational device exemption (IDE) for use in the regions of the U.S. with outbreaks of chikungunya and dengue virus.
The plasma inactivation system uses the light-activated compound amotosalen to reduce the risk of transfusion-transmitted infections. The plasma system has been available in the U.S. under an IDE for the treatment of convalescent plasma collected from Ebola disease survivors for passive immune therapy (see BioCentury Extra, Dec. 17).
Roche (SIX:ROG; OTCQX:RHHBY) fell CHF18.30 to CHF270.40 on Friday after reporting disappointing data from a Phase III trial of breast cancer drug Kadcyla ado-trastuzumab emtansine and discontinuing a Phase III study of gantenerumab to treat prodromal Alzheimer's disease (AD).
In the Phase III MARIANNE trial, topline PFS data showed Kadcyla monotherapy and Kadcyla plus Perjeta pertuzumab both demonstrated non-inferiority, but not superiority, to Herceptin trastuzumab plus taxane chemotherapy when used as first-line therapies. Roche's Genentech Inc. unit developed all three drugs.
Kadcyla is approved in the U.S., EU and Japan as second-line treatment for metastatic HER2-positive breast cancer. The humanized mAb against epidermal growth factor receptor 2 (EGFR2; HER2) is linked to a DM1 cytotoxic agent from ImmunoGen Inc. (NASDAQ:IMGN). Roche's Genentech Inc. unit has rights to use ImmunoGen's TAP antibody-conjugate technology under a 2000 deal.
Cowen analyst Boris Peaker lowered his 2025 peak sales estimate for Kadcyla to $1.8 billion from $7.8 billion on the news. ImmunoGen plunged $4.74 (44%) to $6.11 on Friday.
Roche said it would discontinue the 799-patient SCarletRoAD trial of gantenerumab to treat prodromal AD in pre-symptomatic patients after the HuCAL-derived human mAb targeting beta amyloid failed an interim futility analysis.
The company said it will continue the Phase III Marguerite RoAD trial, which is evaluating the candidate in about 1,000 patients with early AD. Roche's gantenerumab partner MorphoSys AG (Xetra:MOR; Pink:MPSYF) lost EUR 9.70 (11%) to EUR 77.02 in Frankfurt on Friday.
Roche spokesperson Tara Iannuccillo said it would be "premature" to comment on the future of crenezumab, an anti-amyloid antibody that missed the co-primary endpoints in the Phase II ABBY trial in mild to moderate AD. There were trends toward treatment effects in patients with milder disease.
Genentech has exclusive, worldwide rights to crenezumab from AC Immune S.A. (Lausanne, Switzerland) under a 2006 deal (see BioCentury, July 28).
Genentech is partnered with not-for-profit Banner Alzheimer's Institute (BAI) under the Alzheimer's Prevention Initiative (API) to study crenezumab in cognitively healthy adults who share a rare genetic mutation that Genentech said typically triggers Alzheimer's symptoms around age 45.
Juno Therapeutics Inc. (NASDAQ:JUNO) gained $11 (46%) to $35 on Friday, its first day of trading, after raising $264.6 million through the sale of 11 million shares at $24 in its IPO. The cancer immunotherapy developer finished the day with a market cap of $2.7 billion (see BioCentury Extra, Dec. 18).
Juno's three most advanced programs -- JCAR017, JCAR015 and JCAR014 -- use autologous T cells expressing chimeric antigen receptors (CARs) specific to CD19. The company intends to start a Phase II trial of JCAR015 in acute lymphoblastic lymphoma (ALL) in mid-2015, and a Phase I/II trial of JCAR017 in non-Hodgkin's lymphoma (NHL) in 2015.
Sage Kelly resigned as managing director and global head of healthcare investment banking at Jefferies & Co. Inc.
Laurence Reid is resigning as CBO of Alnylam Pharmaceuticals Inc. (NASDAQ: ALNY), effective Dec. 31. He will join Third Rock Ventures as an entreupreneur-in-residence and is expected to become CEO of a portfolio company in the future.
Healthcare private equity firm Linden Capital named Ron Labrum an operating partner. Most recently, Labrum was president and CEO of device company Fenwal Inc., which was acquired by Fresenius SE & Co. KGaA (Xetra:FRE; OTCQX:FSNUY) in December 2012.
In a memo outlining priorities for FDA's Center for Drug Evaluation and Research, center director Janet Woodcock disclosed an initiative to "rapidly re-evaluate our regulation of drug advertising and promotion in light of current jurisprudence around the First Amendment."
The agency has lost a string of cases involving free speech rights since 1999. In December 2012, the U.S. Court of Appeals for the Second Circuit ruled that FDA violated a drug salesman's free speech rights when it prosecuted him for discussing a drug's off-label uses (see BioCentury, Dec. 10, 2012).
Among many other priorities, Woodcock listed patient engagement. In addition to public meetings with patients, the center is developing a quantitative benefit-risk assessment template that incorporates burden of disease. Woodcock noted that patients are "experts" on chronic diseases, and described the challenges in integrating their views into regulations of thousands of diseases.
She said the agency considers "how to meaningfully collect that knowledge, in a rigorous manner, given that there is a spectrum of opinions and a spectrum of disease burden in any given disease," and called for patient and professional groups to submit draft guidance documents (see BioCentury Extra, June 25).
Woodcock also said CDER is evaluating the effects of requiring cardiovascular studies in some chronic indications such as diabetes and obesity, and is evaluating approaches for adding indications for targeted cancer therapies.
She reiterated CDER's chronic inability to recruit staff. The center has more than 600 staff vacancies, including a number of senior positions (see BioCentury, April 7).
William Dunn has been promoted to director of FDA's Division of Neurology Products (DNP) from acting director. He takes over the permanent role that had not been filled since longtime division director Russell Katz announced in January 2013 that he planned to retire.
Dunn inherits a regulatory space that is challenged by the need to identify new clinical endpoints and ways to quanitify benefit and risk for diseases that are both clinically and politically urgent (see BioCentury, Feb. 11, 2013).
He joined the agency in 2005 as a medical officer in the division. He became acting deputy director in 2013 and was elevated to acting director in January this year. Dunn previously served as a clinician in the stroke diagnostics and therapeutics section of the National Institute of Neurological Disorders and Stroke (NINDS).
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