Biomarin wins FDA nod for phenylketonuria drug | FDA fast-tracks Krystal's rare skin blistering disorder therapy | AcelRx's pain management drug accepted for FDA review
May 25, 2018
FDLI SmartBrief
News for the food and drug law community
Drugs & Biologics
Biomarin wins FDA nod for phenylketonuria drug
The FDA granted approval to Biomarin Pharmaceutical's Palynziq, or pegvaliase-pqpz, for use in reducing blood phenylalanine concentrations in adult patients with phenylketonuria, whose blood Phe concentrations is higher than 600 micromo/L and is uncontrolled. The decision was supported by data from a late-stage trial in which the drug reduced blood Phe levels of patients versus placebo use.
Rare Disease Report (5/24) 
FDA fast-tracks Krystal's rare skin blistering disorder therapy
Krystal Biotech's KB103 was granted fast-track designation by the FDA. The drug candidate is in a Phase I/II study for the treatment of dystrophic epidermolysis bullosa.
Seeking Alpha (5/24) 
AcelRx's pain management drug accepted for FDA review
The FDA accepted for review AcelRx Pharmaceuticals' Dsuvia, or sufentanil sublingual tablet, 30 mcg, with a PDUFA date of Nov. 3. Dsuvia is being developed for adult patients to manage moderate-to-severe pain under medically supervised settings.
Seeking Alpha (5/24) 
Bio's sBLA for hereditary factor X deficiency drug accepted by FDA
Bio Products Laboratory's supplemental biologics license application for Coagadex, which is being developed as a prophylactic treatment for patients with hereditary factor X deficiency who are younger than 12, was accepted by the FDA. The application was backed by results from a late-stage prospective study of the candidate in which investigators rated it excellent for all study subjects.
MD Magazine online (5/24) 
Merck's Keytruda shows positive results for squamous NSCLC
Merck announced Wednesday that recent study outcomes show that its cancer drug Keytruda increased survival times and decreased recurrence in untreated patients with squamous non-small cell lung cancer, results already seen in other forms of lung cancer. Merck plans to present expanded data at the American Society of Clinical Oncology meeting early next month.
FiercePharma (5/23) 
FDA turns down Recro's nonopioid pain shot
The FDA ruled that Recro Pharma's nonopioid shot IV meloxicam did not meet expectations on pain relief despite the statistically significant outcomes it showed pertaining to its main goals in late-stage trials. The agency also cited issues on the chemistry, manufacturing and controls related to certain data provided by Recro in its application.
Reuters (5/24) 
Global Focus
Sandoz gets EU approval for biosimilar Zessly
Novartis unit Sandoz's Zessly, or biosimilar infliximab, was approved by the European Commission for all of the same indications for which its reference medicine, Merck and Johnson & Johnson's Remicade, has been cleared -- including Crohn's disease, ankylosing spondylitis, plaque psoriasis, psoriatic arthritis, rheumatoid arthritis and ulcerative colitis.
PharmaTimes online (UK) (5/24) 
Chiesi's alpha-mannosidosis drug turned down by NICE
The National Institute for Health and Care Excellence recommended against the use of Chiesi's Lamzede, or velmanase alfa, in patients with alpha-mannosidosis. The cost regulator ruled that trial evidence showed the nature and size of the clinical benefits are uncertain in the short and long term, and the drug's benefit did not meet cost-effectiveness standards.
PharmaTimes online (UK) (5/24) 
Food & Dietary Supplements
Listeria risk triggers recall of frozen broccoli cuts
A recall was issued by Stop & Shop for its SB Cut Broccoli because it could be contaminated with listeria monocytogenes. The recalled product bears the UPC number 068826700926 and the "best-by date of March 15, 2020, and was distributed to Stop & Shop stores in Connecticut, Massachusetts, New Jersey, New York and Rhode Island.
Food Poisoning Bulletin (5/24) 
Medical Devices
Mauna Kea's Cellvizio cleared by FDA for neurosurgical use
Mauna Kea Technologies has secured 510(k) clearance from the FDA for its laser endomicroscope Cellvizio 100 series F400 and F800 with the CranioFlex, a new Confocal Miniprobe, for use in neurosurgery.
Seeking Alpha (5/24) 
Food and Drug Law Institute Highlights
Submit your work: 2018 Austern Writing Competition
Submit your work: 2018 Austern Writing Competition
Student submissions wanted for FDLI's H. Thomas Austern Writing Competition! Winners receive up to $750, and top papers will be considered for publication in the Food and Drug Law Journal. Accepting entries through June 11. Submit your work.
Update Magazine feature -- China: Breaking Down the "Great Wall"?
Update Magazine feature -- China: Breaking Down the "Great Wall"?
Xin Tao of Hogan Lovells reports for FDLI's Update Magazine on recent changes to the Chinese regulatory scheme aimed at facilitating market entry for pharmaceuticals. Developments to watch include changes in the approval process, patent rights and overseas inspections. Read more.
Learn more about FDLI:
About | Programs | Resources | Engage | Career
Editor's Note
SmartBrief will not publish Monday
In observance of Memorial Day in the US, SmartBrief will not publish Monday. Publication will resume Tuesday.
SmartBrief Privacy Policy
SmartBrief has updated its Privacy and Cookie Policy. Here are the key things for you to note: Our Privacy and Cookie Policy has been rewritten to make it easier to understand and to address new EU requirements. The policy outlines how we collect, use, share and protect your personal data. It also includes details on how you can make choices about your information and the measures we've put in place to keep your information secure. We encourage you to review our new policy at
powered by
Director, Office of Surveillance and Compliance
Associate Director, Corporate Counsel - Regulatory and Commercial
REGENERON - Tarrytown, NY
Food and Drug Associate
Adjunct Faculty - Regulatory Affairs and Health Policy
Only in growth, reform and change, paradoxically enough, is true security to be found.
Anne Morrow Lindbergh,
writer and aviator
Sign Up
SmartBrief offers 200+ newsletters
Learn more about the SmartBrief audience
Subscriber Tools:
Contact Us:
Jobs Contact  -
Advertising  -  Sarah Hostetler
Editor  -  Lisa Gough
Mailing Address:
SmartBrief, Inc.®, 555 11th ST NW, Suite 600, Washington, DC 20004
© 1999-2018 SmartBrief, Inc.®
Privacy Policy (updated May 25, 2018) |  Legal Information