Bio Roundup: PDUFA Progress, BCRA Redux, CAR-T Thumbs Up, & More

Xconomy National — 

Government and industry are rife with awkward acronyms. Take PDUFA, for example.

First passed in 1992, the Prescription Drug User Fee Act allows the FDA to collect fees from biopharmaceutical companies when they apply for drug approval; those funds are designated for the regulator’s drug evaluation work. Congress must reauthorize the law every five years and it’s up for renewal this year. This week, the House of Representatives passed legislation to renew PDUFA. As in previous years, lawmakers approved the measure with bipartisan support. It now advances to the Senate.

Moving on to legislation that does not enjoy bipartisan support, Senate Republicans on Thursday released a revamped bill to repeal the Affordable Care Act. According to The New York Times, the new version of the Better Care Reconciliation Act aims to “hold down insurance costs for consumers while keeping a pair of taxes on high-income people that they had planned to eliminate.” Even so, the new measure was met with alternative proposals and resistance from some Republicans who opposed the bill’s Medicaid cuts, The Times reports.

In other news, the past week saw the approval of a new sickle cell disease drug, a path forward for a Fabry disease drug, and an approval recommendation for a new kind of cancer immunotherapy. Let’s get to the roundup.


—Johnson & Johnson (NYSE: JNJ) subsidiary Janssen Biotech received FDA approval for guselkumab (Tremfya), an antibody drug for moderate to severe plaque psoriasis that will compete with AbbVie (NYSE: ABBV) blockbuster adalimumab (Humira).

—Amicus Therapeutics (NASDAQ: FOLD) is preparing to file for FDA approval of Fabry disease drug migalastat after the regulator dropped a requirement that the Cranbury, NJ, biotech complete a Phase 3 trial. Fabry disease is a rare inherited disorder that causes an enzyme deficiency that can progress to organ damage.

—Arena Pharmaceuticals (NASDAQ: ARNA) announced that early Phase 2 results for its pulmonary arterial hypertension drug ralinepag met the trial’s main goal—news that sent the San Diego biotech’s shares up by nearly 50 percent.

—Emmaus Medical of Torrance, CA, received FDA approval for L-glutamine (Endari), the first new drug to treat sickle cell disease in nearly 20 years.

—An external advisory panel gave the FDA a unanimous thumbs-up on CAR-T cancer therapies on July 12. The recommendation paves the way toward a possible FDA approval for the Novartis (NYSE: NVS) treatment for children and young adults with a severe form of leukemia, which would be the first for an immunotherapy of this kind.


—The FDA rejected an eye drug delivery device from Ocular Therapeutix (NASDAQ: OCUL), citing manufacturing problems found in May. If those problems are not resolved, the FDA said it could not approve the Bedford, MA, company’s application.

—Trabodenoson, a glaucoma drug from Lexington, MA-based Inotek Pharmaceuticals (NASDAQ: ITEK), failed a Phase 2 test marking the drug’s second clinical trial failure this year. The company says it will consider “strategic options.”

—Meanwhile, Galena Biopharma (NASDAQ: GALE) said it is cutting staff and conserving cash as it weighs its own strategic options that could include a deal for the company or its drugs. The San Ramon, CA-based biotech is enrolling breast cancer patients in a Phase 2b clinical trial testing lead drug Nelipepimut-S (NeuVax).

David Dvorak unexpectedly stepped down as CEO of Zimmer Biomet (NYSE: ZBH). The news that came as the Warsaw, IN, medical device company released preliminary second quarter results suggesting weaker earnings.


—Shire (NASDAQ: SHPG) got a legal win in its efforts to fend off potential competition from Roche hemophilia A drug emicizumab. A German court issued a preliminary injunction barring Roche from stating that adverse events in its studies occurred in four patients who received high doses of Shire’s drug—claims Shire says are unsupported.

—The hemophilia community had more reasons to pay attention to Germany. The International Society on Thrombosis and Haemostasis met in Berlin, where Spark Therapeutics (NASDAQ: ONCE) presented interim clinical trial data for its hemophilia B gene therapy SPK-9001.

—At the same conference, San Rafael, CA-based BioMarin (NASDAQ: BMRN) presented clinical updates for its hemophilia A gene therapy, BMN 270.


Evelo Biosciences raised $50 million in funding that the Cambridge, MA, biotech plans to use to prepare microbiome drugs for clinical trials in cancer and immune system disorders.

—Cambridge-based cancer drug developer Neon Therapeutics entered a research collaboration with CRISPR Therapeutics (NASDAQ: CRSP) to explore ways to combine their respective technologies to develop new T cell therapies.

—Sanofi (NYSE: SNY) agreed to pay $650 million up front to acquire Protein Sciences, a Meriden, CT, biotech that uses genetic engineering and cell culture to make flu vaccines.

E-Scape Bio’s Series A round expanded to $63 million, which will support the South San Francisco, CA-based biotech’s work developing Alzheimer’s and Parkinson’s disease drugs.

—Natick, MA-based Allurion Technologies raised $27 million to finance a clinical trial for its non-surgical gastric balloon for weight loss.

—DNA-synthesis company Twist Bioscience of San Francisco announced two separate partnerships: one with a synthetic biology firm called Synbio Technologies, and the other with a sequencing and molecular biology company, Quintara Biosciences. Twist will produce DNA snippets that its partners will then use to manufacture longer genes or gene fragments—part of a larger push in synthetic biology to make longer stretches of DNA faster and at lower costs.

David Holley and Corie Lok contributed to this report.

Photo by Flick user Kevin Dooley via a Creative Commons license.