The Daily Biotech Pulse: FDA Orphan Drug Tags For Neurocrine, Editas Candidates; Sale of BridgeBio's PRV; RMAT Designation For PolarityTE's Product

Zinger Key Points
  • The FDA has granted Orphan Drug Designation to Neurocrine Biosciences Inc's valbenazine for Huntington's disease.
  • Valbenazine is a selective vesicular monoamine transporter 2 inhibitor being investigated as a once-daily treatment in adults with chorea associated with HD.

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

Stocks In Focus

Switzerland Approves Moderna's COVID-19 Shot For Kids 6-11

The Swiss drug regulatory authority has approved Moderna Inc's MRNA COVID-19 vaccine for children ages 6-11. 

The approval is for the vaccine's two-dose series of 50 micrograms per dose, Moderna added.

Shares were up 6.72% at $138.92 Friday. 

Neurocrine's Huntington's Candidate Secures FDA Orphan Drug Tag

The FDA has granted Orphan Drug Designation to Neurocrine Biosciences Inc's NBIX valbenazine for Huntington's disease (HD). 

The treatment of chorea associated with HD is within the scope of this Orphan Drug Designation. 

Valbenazine is a selective vesicular monoamine transporter 2 inhibitor being investigated as a once-daily treatment in adults with chorea associated with HD.

Editas Gene-Editing Thalassemia Treatment Gets FDA Orphan Drug Status

Editas Medicine Inc's EDIT EDIT-301, an investigational gene-editing medicine for beta-thalassemia, has received FDA Orphan Drug Designation.

The FDA previously granted Rare Pediatric Disease designation to EDIT-301 for beta-thalassemia and sickle cell disease.

EDIT-301 is being investigated in a clinical study in severe sickle cell disease patients. Editas expects to initiate a Phase 1/2 study of EDIT-301 in patients with transfusion-dependent beta-thalassemia in 2022.

CSL's Vifor Buyout Hits Regulatory Delay

CSL Ltd's CSLLY $11.7-billion buyout of Vifor Pharma AG GNHAY will have to wait a few more months.

While CSL previously expected to close the acquisition this June, the regulatory approval process is taking longer than expected. Some antitrust authorities have approved the transaction, but others remain outstanding, Vifor said in a statement.

Bristol Myers Sells New York Biologics Facility

LOTTE Corporation has agreed to purchase Bristol Myers Squibb Co's BMY manufacturing facility in East Syracuse, New York. 

The East Syracuse site will serve as the LOTTE Center for North America Operations for LOTTE's new biologics contract development and manufacturing organization business in the U.S.

The companies anticipate completing the transaction by the second half of 2022.

Deal terms were not disclosed.

Statera Biopharma, Lay Sciences Ink License Pact For IgY Products

Statera Biopharma STAB intends to enter into a non-binding term sheet for a strategic partnership with Lay Sciences Inc to license worldwide manufacturing rights to certain Lay Sciences' IgY products containing avian antibodies as an active ingredient.

Lay Sciences would receive an upfront payment including equity and cash and would also have the potential to earn royalties and milestones associated with the development of IgY in gastrointestinal and upper respiratory indications.

Specific deal terms were not disclosed.

Shares were up 19.4% at 27 cents during the premarket session.

BridgeBio Pharma Sells Priority Review Voucher For $110M

BridgeBio Pharma Inc BBIO has agreed to sell its rare pediatric disease Priority Review Voucher for $110 million to an undisclosed purchaser.

BridgeBio was awarded the voucher when its affiliate, Origin Biosciences Inc., received approval of Nulibry (fosdenopterin) for injection as the first therapy to reduce the mortality risk in patients with molybdenum cofactor deficiency Type-A. 

BridgeBio has amended its existing senior secured credit facility, extending the interest-only period by two years and principal repayment to Nov. 17, 2026. 

Lantheus Starts Dosing In Imaging Agent Study to Monitor PD-L1 Expression In Lung Cancer Patients

Lantheus Holdings Inc LNTH has dosed the first patient in a Phase 2 trial evaluating NM-01 for its potential to identify patients who will respond to checkpoint inhibitor therapies.

NM-01 is a technetium 99m SPECT imaging agent used to assess PD-L1 expression in cancer cells.

Shares are rising 7.67% at $64.17 during the premarket session.

PolarityTE Shares Jump On FDA's RMAT Tag For SkinTE

The FDA has granted Regenerative Medicine Advanced Therapy (RMAT) designation to PolarityTE Inc's PTE SkinTE under the company's open IND. 

RMAT designation is a dedicated program designed to expedite the drug development and review processes for promising regenerative medicine products.

Shares are up 42.9% at 21 cents during the premarket session.

On The Radar


The earnings list presented is not comprehensive. Click here to access Benzinga's earnings calendar for the complete schedule.

PLx Pharma Inc PLXP: Before Market Open.

Better Therapeutics Inc BTTX: Before Market Open.

Biofrontera Inc BFRI: Before Market Open.

ProPhase Labs Inc PRPH: Before Market Open.

Longeveron Inc LGVN: Before Market Open.

EQRx Inc EQRX: Before Market Open.

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