Clovis Oncology Inc. (NASDAQ:CLVS) gained $4.93 (27%) to $23.03 on Tuesday after it said FDA accepted and granted Priority Review to an NDA seeking accelerated approval of rucaparib (CO-338) as monotherapy in patients with germline or somatic BRCA-mutated advanced ovarian cancer who have received multiple prior chemotherapies. Its PDUFA date is Feb. 23, 2017.
The oral inhibitor of PARP-1 and PARP-2 has breakthrough therapy designation from FDA for the indication (see BioCentury Extra, April 6, 2015).
Clovis also disclosed updated data from two Phase II studies in which a total of 106 patients with BRCA-mutated ovarian cancer received the therapy. The overall response rate (ORR) across both studies was 54%, including a 9% complete response rate and 45% partial response rate. The median duration of response was 9.2 months. One study was limited to platinum-sensitive patients, while the second study included platinum-sensitive, platinum-resistant and platinum-refractory patients.
Clovis has exclusive, worldwide rights to rucaparib from Pfizer Inc. (NYSE:PFE). Rucaparib became Clovis' lead program in May after the company shifted its resources away from non-small cell lung cancer (NSCLC) candidate rociletinib (CO-1686) (see BioCentury Extra, May 5).
The company's shares remain well below their 52-week high of $116.75, reached in September 2015.
Australia's Pharmaceutical Benefits Advisory Committee (PBAC) has given pharmacists the authority to substitute the biosimilar Brenzys etanercept for its reference product, autoimmune drug Enbrel etanercept.
Merck & Co. Inc. (NYSE:MRK) holds rights to Brenzys in Australia under a partnership with Samsung Bioepis, a JV between Biogen Inc. (NASDAQ:BIIB) and Samsung Group (Seoul, South Korea). Pfizer Inc. (NYSE:PFE) holds Enbrel's rights outside the U.S. and Canada under a deal with Amgen Inc. (NASDAQ:AMGN).
PBAC said Brenzys "could be marked as equivalent" to Enbrel on the Australian Pharmaceutical Benefits Scheme (PBS), allowing the biosimilar's substitution to treat all indications for which both drugs are approved. PBAC noted that a prescriber may choose to not permit substitution of the biosimilar.
Brenzys is also approved in South Korea, and is marketed in Europe as Benepali.
In July, Australia's Therapeutic Goods Administration (TGA) approved Brenzys to treat rheumatoid arthritis, psoriatic arthritis, plaque psoriasis, ankylosing spondylitis and non-radiographic axial spondyloarthritis.
Clarus co-founder and Managing Director Michael Steinmetz passed away Aug. 6 from heart failure due to familial dilated cardiomyopathy, a heritable condition.
In a notice posted on its website, Clarus said Steinmetz's family has created a not-for-profit foundation to fund research led by his son Lars Steinmetz seeking a cure for the disease. Lars Steinmetz is a genetics professor and co-director of the Genome Technology Center at Stanford University.
Sophiris Bio Inc. (NASDAQ:SPHS) raised $26 million through the sale of 6.5 million units at $4 in a follow-on underwritten by Piper Jaffray and Maxim. Each unit includes a share plus a warrant to acquire 0.75 shares. The company proposed the offering after market close on Monday, when its share price was $5.82. Sophiris sank $1.93 (33%) to $3.89 on Tuesday.
The company plans to run a Phase IIb study of topsalysin (PRX302) to treat localized prostate cancer. It hopes to have data by 2H17. The therapy is a recombinant proaerolysin protein activated by prostate-specific antigen (KLK3; PSA).
The California Assembly passed the Right To Try Act (AB-1688), which would allow drug manufacturers and physicians to provide eligible terminally ill patients with investigational therapies that have completed a Phase I trial but have not been approved by FDA. The California Senate passed the bill last week.
If the bill reaches Gov. Jerry Brown's desk by Sept. 1, he will have until Sept. 30 to sign or veto it.
In a message to legislators as he vetoed a similar right-to-try bill last year, Brown said FDA's compassionate use program should be given "a chance to work" before the state authorizes an alternative pathway (see BioCentury Extra, Oct. 12, 2015).
In June, FDA clarified its expanded access policies and issued a final version of a shortened form for physicians to request drugs for compassionate use (see BioCentury Extra, June 2).
Assembly member Ian Calderon (D) sponsored the bill. Calderon spokesperson Lerna Shirinian told BioCentury that while the new form reduces physicians' obligations, FDA's revisions to the program neither expedite the expanded access process for manufacturers nor reduce patients' waiting periods while FDA decides whether to provide drugs under the program.
In a draft evidence report evaluating non-small cell lung cancer drugs, the Institute for Clinical and Economic Review found that three marketed tyrosine kinase inhibitors' cost-effectiveness appeared to fall within "commonly-accepted thresholds." ICER also found that three mAbs against PD-1 or PD-L1 were outside the range of cost-effectiveness, but stressed that variability in those drugs' overall survival (OS) and progression-free survival (PFS) benefits led to uncertainty about the cost-effectiveness estimates used in ICER's analysis.
ICER evaluated the TKIs Gilotrif afatinib, Tarceva erlotinib and Iressa gefitinib compared to cisplatin plus Alimta pemetrexed as first-line therapies for EGFR-positive NSCLC. It compared anti-PD-L1 mAb Tecentriq atezolizumab and anti-PD-1 mAbs Opdivo nivolumab and Keytruda pembrolizumab vs. docetaxel as second-line therapies for EGFR-negative NSCLC.
The institute estimated incremental cost-effectiveness ratios for the three TKIs of $110,000-$130,000 per quality-adjusted life year (QALY) gained vs. the comparator. Boehringer Ingelheim GmbH (Ingelheim, Germany) markets Gilotrif. The Genentech unit of Roche (SIX:ROG; OTCQX:RHHBY) and Astellas Pharma Inc. (Tokyo:4503) market Tarceva, and AstraZeneca plc (LSE:AZN; NYSE:AZN) markets Iressa.
For the mAbs, ICER estimated incremental cost-effectiveness ratios of $208,000-$250,000 per QALY gained vs. the comparator. It said the drugs' cost-effectiveness "ranged similarly in scenario analyses" based on levels of PD-L1 expression. Bristol-Myers Squibb Co. (NYSE:BMY) markets Opdivo, Merck & Co. Inc. (NYSE:MRK) markets Keytruda and Roche markets Tecentriq atezolizumab.
ICER said value-based price benchmarks will be provided as part of its full evidence report, due in late September.
Comments on the draft report are due Sept. 16. ICER's Midwest Comparative Effectiveness Public Advisory Council will hold a public meeting on Oct. 20 to discuss the assessments.
FDA's Center for Devices and Radiological Health and Center for Biologics Evaluation and Research released final guidance on the use of patient preference information to inform regulatory decisions concerning medical devices, including premarket approvals and de novo submissions.
The agency released draft guidance on the subject in May 2015. Spokesperson Deborah Kotz told BioCentury the new guidance clarifies how stakeholders can submit patient preference information to FDA, includes a section about adding relevant information to decision summaries, and updates recommendations to manufacturers about device labeling (see BioCentury Extra, May 13, 2015).
Both the draft and final guidances said patient preference information can be used to help identify the most important benefits and risks of using a technology, to assess the relative importance of those benefits and risks and how patients assess the balance among them, and to help understand the heterogeneity or distribution of patient preferences concerning different treatment or diagnostic options.
CDRH has pioneered FDA's efforts to incorporate patient preferences in regulatory decisions (see BioCentury, Feb. 23, 2015).
In letters to Mylan N.V. (NASDAQ:MYL) CEO Heather Bresch, U.S. Sens. Chuck Grassley (R-Iowa) and Richard Blumenthal (D-Conn.) expressed concern that recent increases in EpiPen's price could limit patients' access to the epinephrine auto-injector to treat severe acute allergic reactions. The lawmakers requested information about access programs, pricing and advertising cost transparency, and changes Mylan has made in the product since acquiring it in 2007.
On Saturday, Sen. Amy Klobuchar (D-Minn.) called on the Senate Judiciary Committee to hold a hearing to investigate EpiPen's price and alleged Mylan is "exploiting a monopoly market advantage" due to reduced competition. On Monday, Klobuchar also asked the U.S. Federal Trade Commission to investigate EpiPen's price increase.
The lawmakers said the price of an EpiPen two-pack has risen to $500 or more, from $100 in 2009.
In a press release on Monday, Mylan highlighted some of its access programs and newer product features. The company said that as more consumers are enrolled in high deductible health plans, they are "bearing more of the cost" of therapies.
Grassley is chairman of the Senate Judiciary Committee. Klobuchar and Blumenthal serve on its antitrust subcommittee, and Klobuchar is the subcommittee's ranking member.
Mylan lost $2.28 to $45.62 on Tuesday, and is down 6% since Friday's close of $48.66.