The Daily Biotech Pulse: FDA Tentatively Approves Avadel's Narcolepsy Drug, European Approval Broadens AstraZeneca's Enhertu in Breast Cancer, Pharma Giants' Earnings

Zinger Key Points
  • Tentative approval confirms the safety profile, clinical efficacy of Lumryz for narcolepsy
  • Incyte's Opzelura is the first and only FDA-approved product for repigmentation in nonsegmental vitiligo.
The Daily Biotech Pulse: FDA Tentatively Approves Avadel's Narcolepsy Drug, European Approval Broadens AstraZeneca's Enhertu in Breast Cancer, Pharma Giants' Earnings

Here's a roundup of top developments in the biotech space over the last 24 hours:

Stocks In Focus

AstraZeneca's Enhertu Wins European Approval For Pretreated Breast Cancer Setting

The European Commission has approved AstraZeneca Plc AZN and Daiichi Sankyo's DSNKY Enhertu as monotherapy for unresectable or metastatic HER2-positive breast cancer who have received one or more prior anti-HER2-based regimens.

Following EU approval, AstraZeneca will pay $75 million to Daiichi Sankyo as a milestone payment.

Ultragenyx's Neurogenetic Disorder Candidate Shows Favorable Tolerability, Clinical Activity

Ultragenyx Pharmaceutical Inc RARE and GeneTx Biotherapeutics LLC announced interim data from the Phase 1/2 study in pediatric patients with a genetically confirmed diagnosis of full maternal UBE3A gene deletion. 

Interim results demonstrate a meaningful improvement in clinical disease and an acceptable safety profile. 

Ultragenyx also announced it has exercised its option to acquire GeneTx and has closed on the acquisition for an upfront payment of $75 million, plus future milestone and royalty payments.

FDA Approves Avadel's Lead Drug For Excessive Sleepiness

The FDA granted tentative approval to Avadel Pharmaceuticals plc's AVDL Lumryz, also known as FT218, for excessive daytime sleepiness or cataplexy in adults with narcolepsy.

Tentative approval indicates that Lumryz has met all required quality, safety, and efficacy standards necessary for approval in the U.S. 

Final approval is pending disposition of U.S. Patent No. 8,731,963. 

Avadel is currently evaluating the long-term safety and tolerability of Lumryz in the open-label RESTORE clinical study.

Shares are up 9.58% at $4.69 during the premarket session.

Bavarian Nordic Secures European Smallpox / Monkeypox Vaccine Order

A non-disclosed country has ordered an additional 1.5 million doses of Bavarian Nordic A/S's BVNRY Imvanex, a non-replicating smallpox vaccine. 

Deliveries will start in the fourth quarter of 2022, but most doses will be delivered during 2023.

Celcuity's Breast Cancer Candidate Gets FDA Breakthrough Therapy Tag

The FDA has granted breakthrough therapy designation to Celcuity Inc's CELC gedatolisib HR+/HER2- metastatic breast cancer whose disease progressed during treatment with a CDK4/6 therapy and a non-steroidal aromatase inhibitor.

Gedatolisib previously received Fast Track designation from the FDA in January 2022.

Shares are up 12.3% at $10.39 during the premarket session.

FDA Approves First Product For Repigmentation In Nonsegmental Vitiligo

The FDA has approved Incyte Corporation's INCY Opzelura (ruxolitinib) cream 1.5% for nonsegmental vitiligo in patients 12 years and older. 

Opzelura is the first and only FDA-approved treatment for repigmentation in patients with vitiligo and the only topical formulation of a Janus kinase inhibitor approved in the U.S. 

Vitiligo is a chronic autoimmune disease characterized by depigmentation of the skin.

Click here to access Benzinga's FDA Calendar.

Apellis's Lead Product For Eye Disorder Under FDA Priority Review

The FDA has granted priority review to Apellis Pharmaceuticals Inc's APLS marketing application seeking approval for intravitreal pegcetacoplan for geographic atrophy secondary to age-related macular degeneration.

The PDUFA date is November 26. The FDA has stated that it is not currently planning to hold an advisory committee meeting to discuss the application.

Apellis plans to submit a marketing application to the European Medicines Agency in 2H of 2022.

Shares are up 24% at $55.51 during the premarket session on Tuesday.

Antengene Inks Research Pact With For Cancer Combo Therapies

Antengene Corporation Limited has entered into a preclinical research collaboration with Celularity Inc CELU.

The companies will evaluate the potential therapeutic synergy combining Antengene's bispecific antibody with Celularity's cryopreserved human placental hematopoietic stem cell-derived natural killer cell therapy platform.

CELU shares are up 7.20% at $3.87 during the premarket session.

Acadia Submits US Application For Rett Syndrome Candidate

Acadia Pharmaceuticals Inc ACAD has submitted a marketing application seeking approval for trofinetide for Rett syndrome in patients two years of age and older.

The FDA has also granted Trofinetide Rare Pediatric Disease designation. With such designation, Acadia expects to be awarded a Priority Review Voucher if the application is approved.

On The Radar

Earnings

Johnson & Johnson JNJ: Before the market open.

Novartis AG NVS: Before the market open.

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