15 March 2019 No. 3946 Scripscrip.pharmaintelligence.informa.com Pharma intelligence | informa time, which is difficult. He also used the office in a “forward-leaning way” not seen often. “He kind of drove the conversation through very good communication exter- nally,” Myers said. “He was able to use a lot of the ideas percolating in the agency, pack- age them and get them moved forward.” Gottlieb pushed FDA to the forefront of the drug pricing debate, a position the agency had not sought in the past. In his first speech to staff after confir- mation, he said that even though the agency cannot set drug prices, it can promote competition. Gottlieb was vocal in chastising the brand industry for blocking generic Credit: FDA Credit: competition, warning that Congress could make more drastic changes if it did not stop employing pay-for-delay and licensing tactics intended to pre- Gottlieb’s Shock Resignation vent patent challenges. (Also see “Got- tlieb: Real Risk Of Congressional Action If Leaves Ripples In The Industry Anti-Competitive Actions Continue” - Pink Sheet, 3 May, 2018.) DERRICK GINGERY [email protected] He also publicly released a list of com- panies thought to be using the Risk Evalu- S FDA Commissioner Scott Gott- being apart from my family for these past ation and Mitigation Strategy system to lieb, one of the most popular and two years and missing my wife and three prevent generic companies from purchas- Uvisible commissioners in decades, young children.” ing samples for testing, although it is not will end his tenure as head of FDA in about clear the move had much impact. (Also a month, having moved the agency in THE PUBLIC COMMISSIONER see “REMS Abuse Website: Celgene, Acte- new directions while also advancing high- Gottlieb may be most remembered for his lion Top List Of Suspected ‘Gamers’” - Pink profile policies. approach to communications. He often Sheet, 18 May, 2018.) Gottlieb amassed a legacy that in- appeared on television and his daily Twit- In addition, Gottlieb blasted the rebate cludes new approaches to drug pricing ter posts complemented the numerous system that he alleged was holding back and opioid policy, as well as record ge- press releases announcing policy changes biosimilar competition for expensive bio- neric and novel drug approval output, and other moves within the agency. Pre- logic drugs. despite running the agency less than vious commissioners tended to avoid the His emphasis on creating generic com- two years. limelight, as well as social media. petition was partially successful. The Gottlieb cited his family in his decision Nancy Myers, president of Catalyst agency set a record for generic drug ap- to leave. In a note to staff, he said “there’s Healthcare Consulting, said in an in- provals in fiscal years 2017 and 2018, al- perhaps nothing that could pull me away terview that Gottlieb was pro-patient, though critics argued that not nearly as from this role other than the challenge of pro-industry and pro-safety at the same CONTINUED ON PAGE 4 BROUGHT TO YOU BY THE EDITORS OF PHARMASIA NEWS, START-UP AND SCRIP INTELLIGENCE AbbVie’s Humira Successor? Starry-Eyed Buy All-Time High Skyrizi set to muscle in on Biogen moves beyond neurology Cell and gene therapy investments competitive psoriasis market (p6) with Nightstar deal (p12) sky rocket in 2018 (p13) IN THIS ISSUE from the editor [email protected] Gene and cell therapies experienced a major investment er’s executives – who have also declared the German boost in 2018, as we report on p13-14 of this issue. With company’s desire to “be at the forefront of the activities all the activity so far this year, the field could well attract in cell and gene therapy” (p21-23). Criticizing govern- even greater sums in 2019. At the same time as venture ments in Europe for supporting a car industry that has capital and public market funds have been rolling in, big “basically been reduced to inventing batteries” while pharma has also been investing, not least through acqui- overlooking the value created by life sciences and cre- sitions like Biogen’s deal for Nightstar Therapeutics and ating a regulatory framework that is hostile to tech- Roche’s for Spark Therapeutics (see p12 and p15). nologies like gene editing, they were equally scathing But for a sector with nearly 1,000 companies in opera- of the UK politicians who called the Brexit referendum, tion, there is still a large question mark over its com- branding their behaviour “highly irresponsible.” mercial viability, since only a few therapies have made it The long shadows of Brexit certainly aren’t making to market, and those that have are generally sold at high it any easier for European biopharma in its long-run- cost in low volumes, with complicated manufacturing. ning attempts to cultivate a more fertile environment Elsewhere in the issue read about the concerns over for early science to put down strong roots, flourish and conditions in Europe for innovation expressed by Bay- mature on the continent where it was seeded. 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ISSN 0143 7690. 2 | Scrip | 15 March 2019 © Informa UK Ltd 2019 Tecentriq Steals Lead Entrectinib’s Bayer’s Labored Ascent Kemal Malik 20 7 11 21 exclusive online content inside: COVER / Gottlieb’s Shock Resignation Leaves Ripples Interview: ViroMed On Pricing, In The Industry Manufacturing World’s First Broadly Used 6 AbbVie’s Skyrizi Poised To Enter Packed Psoriasis Market Gene Therapy 7 Roche’s Tecentriq Steals Lead In Triple-Negative JUNG WON Shin [email protected] Breast Cancer 8 J&J Foresees Broad Insurance Coverage For Groundbreaking Spravato Nasal Spray 10 Same Drug, Two Versions And Prices, This Time For Lilly’s Humalog 11 Roche’s EU Accelerated Assessment Bid For Tumor Agnostic Entrectinib Backfires 12 Biogen Targets Ophthalmology As Emerging Growth Area With Nightstar Buy 13 Gene Therapy Investment At All Time High, Commercialization Challenges Noted Despite ongoing debate over the sometimes staggeringly high cost of gene therapies, ViroMed Co. Ltd. does not ap- 15 How Roche Won The Bidding Battle For Spark pear too concerned about the likely pricing of its lead as- set in this field, the non-viral therapy VM202. 16 Pro Vs. Con: A Side-By-Side Look At Investor Concerns, Bristol’s Defenses Of Celgene Deal While companies in the sector are considering various approaches, such as installment payments, to help facili- 17 Concert Says Phase II Alopecia Drug Could Beat Pfizer’s tate market access to their pricey products, the South Ko- Candidate To Market rean biotech is looking at pricing the product at a more af- fordable level, especially given that it is targeting diseases 18 Allergan Endures Another R&D Setback With Rapastinel with relatively large patient populations. Failing Three Pivotal Studies The firm is slated to announce later this year top-line re- sults from the first Phase III study with VM202 in its most 20 Alnylam Ramps Up Commercial Planning For Givosiran advanced indication, painful diabetic peripheral neuropa- Based On Phase III Data thy (PDPN). “The latest gene therapies are announcing prices of 21 Bayer Bosses Beg Risk-Averse Europe To Rethink more than a million dollars and this is becoming a social issue. Our therapy targets at least a one million [patient] 22 Pipeline Watch population in the US alone, so we have to find the right 23 Appointments price level that is appropriate for the target disease and the patient populations,” Jehee Suh, executive director of global business strategy at ViroMed, told Scrip in a recent exclusive interview in Seoul. Published online 28 February 2019 @PharmaScrip /scripintelligence To read the rest of this story go to: https://bit.ly/2NVFnna /scripintelligence /scripintelligence scrip.pharmaintelligence.informa.com 15 March 2019 | Scrip | 3 HEADLINE NEWS CONTINUED FROM PAGE 1 the regulatory framework for approv- ing on several important topics for public many of those products were reaching the ing novel technologies, including gene health,” said Sharfstein, who now is vice market. (Also see “US FDA Sets Generic Ap- therapies,” the brand drug trade group dean for public health practice and com- proval Record, But Generic Sponsors Aren’t said. “His efforts have made a meaning- munity engagement at the Johns Hop- Celebrating” - Pink Sheet, 12 Oct, 2018.) ful impact for patients in need of innova- kins Bloomberg School of Public Health. At the same time, the agency also ap- tive medicines.” “These included the harms of nicotine and proved a record number of novel drugs in Gottlieb also oversaw several policy tobacco, the importance of lowering drug 2018, which for the first time comprised changes intent on fighting the opioid prices through generic competition, and mostly orphan products.